A Wisconsin boy with a uncommon illness that causes his pores and skin to blister on the slightest contact has now been recognized with most cancers.

Charlie Knuth, 13, from Darboy, was adopted by his mother and father after he was deserted on the hospital as a child, reported WFRV.

He suffers from epidermolysis bullosa (EB), a uncommon genetic tissue dysfunction that causes the pores and skin to blister and burst, leaving uncooked sores which might be inclined to infections.

Charlie has lived most of his life wrapped in bandages and has to take particular baths on daily basis to deal with his sores and maintain them from getting contaminated.

However the teenager is now going through a brand new battle after being recognized earlier this 12 months with lymphoma, a most cancers of the immune system.

Charlie Knuth, 13, from Darboy, Wisconsin, was born with a uncommon pores and skin dysfunction. Pictured: Charlie, proper, along with his father, Kevin

The dysfunction, often known as epidermolysis bullosa, causes the pores and skin to blister and burst on the slightest contact and leaves uncooked sores. Pictured: Charlie within the hospital)

Victims of EB are lacking sort VII collagen, a protein that enables the highest layer of pores and skin to bind with the underside layers.

The slightest motion can causes the pores and skin to instantly and constantly fall off.

The dysfunction could be very uncommon, and is estimated to happen in 20 newborns per a million reside births within the US, based on Stanford Kidss Hospital.

About 87 % of youngsters born with EB die throughout their first 12 months of life.

There isnt any remedy for EB so remedy goals at stopping blisters from changing into contaminated.

Charlies mom, Trisha Knuth, stated she and his father, Kevin, have tried a number of measures to assist deal with her son, together with lotions, lotions and gloves.

He is additionally undergone two stem cell transplants, during which new sheets of pores and skin grown and graft over the injuries.

In 2017, Charlie underwent surgical procedure to revive the usage of his palms, which had been degenerating because of his situation.

With no surgical procedure, his palms can be lined in scar tissue.

The scar tissue will really construct up between all the net areas between every finger, and the palms, and develop proper excessive of the hand so persons are left with simply nothing however mitts, Trisha advised WFRV.

In keeping with the station, throughout the surgical procedure, pores and skin was taken from Charlies thighs to make use of on his palms.

Titanium rods had been additionally inserted intoevery of his fingers, and saved there for 5 weeks, to stop them from curling into his palms.

Charlie (left and proper) was deserted at a hospital earlier than being adopted by his present mother and father. Earlier this month, he was recognized with lymphoma, a most cancers of immune system cells

His mother and father stated theyre touring to Minnesota to determine what stage his most cancers is at and what remedy hell bear. Pictured: Charlie, proper, along with his mom, Trisha

On Wednesday, Trisha posted on Fb that her son was recognized with lymphoma.

Lymphoma is most cancers that begins within the lymphocytes, that are immune system cells that struggle an infection.

There are two kinds of the most cancers, Non-Hodgkins and Hodgkins, nevertheless its not clear which kind Charlie has.

Indicators and signs embody swelling of the lymph nodes, fever, fatigue, shortness of breath and sudden weight reduction.

Therapy varies and may embody chemotherapy, radiation remedy and immunotherapy.

Its estimated that 82,310 folks will likely be recognized with lymphoma in 2019 and that 20,970 will die, based on the American Most cancers Society.

Within the Fb put up, Trisha wrote: My head is spinning and my coronary heart is breaking. My candy boy.

Charlies mom added the household will likely be touring to Minnesota so his most cancers can get staged and so they can assess remedy choices.

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Boy abandoned at the hospital after being born with butterfly skin disease diagnosed with cancer - Herald Publicist

Skin Stem Cells Comments Off on Boy abandoned at the hospital after being born with butterfly skin disease diagnosed with cancer – Herald Publicist | December 18th, 2019

Aspen Neuroscience, a new biotech company, has raised $6.5 million to develop cell therapies for Parkinsons disease using patients own cells.

The company was co-founded by renowned stem cell scientists Jeanne F. Loring, PhD, and Andres Bratt-Leal, PhD, and initially supported by Summit for Stem Cell, a non-profit organization that provides a variety of services for Parkinsons patients.

Parkinsons hallmark motor symptomsinclude tremor, slowness of movement (bradykinesia), stiffness (rigidity), uncontrollable movements (dyskinesia), and poor balance.

As the disease progresses, patients typically need to gradually increase their dopaminergic therapeutic dose for maximum benefit. Even after that they might sometimes experience reappearance or worsening of symptoms due to diminishing effects of dopaminergic therapy, known was off periods.

Importantly, dopaminergic therapy is delivered to areas of the brain other than the striatum, a key motor control region severely affected in Parkinsons disease. Because of the therapys off-target behavior, patients also may experience side effects such as hallucinations or cognitive impairment.

Aspen wants to combine its expertise in stem cell biology, genomics and neurology and develop the first autologous (self) stem cell-based therapy for Parkinsons disease.

In this type of cell therapy, a patients own cells (usually skin cells) are reprogrammed back into a stem cell-like state, which allows the development of an unlimited source of almost any type of human cell needed, including dopamine-producing neurons, which are those mainly affected by this disorder.

Because these cells are derived from patients, they do not carry the risk of being rejected once re-implanted, eliminating the need for immunosuppressive complementary therapies, which carry serious side effects such as infections and possibly limiting therapeutic potential.

In theory, replacing lost dopaminergic neurons with new stem cell-derived dopamine-producing ones could potentially ease or reverse motor symptoms associated with the disease.

Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinsons disease, Howard J. Federoff, MD, PhD, Aspens CEO, said in a press release.

We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist, Federoff said.

The companys lead product (ANPD001) is undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic Parkinsons disease. Aspen experts also are developing a gene-editing treatment (ANPD002) for familial forms of Parkinsons, starting with the most common genetic variant in the GBAgene, which provides instructions to make the enzyme beta-glucocerebrosidase.

The new seed funding round was led by Domain Associates and Axon Ventures, with additional participation from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32, according to the press release.

With over three years of experience in the medical communications business, Catarina holds a BSc. in Biomedical Sciences and a MSc. in Neurosciences. Apart from writing, she has been involved in patient-oriented translational and clinical research.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Aspen Neuroscience Receives $6.5M for Parkinson's Stem Cell Therapy - Parkinson's News Today

Skin Stem Cells Comments Off on Aspen Neuroscience Receives $6.5M for Parkinson’s Stem Cell Therapy – Parkinson’s News Today | December 16th, 2019

LOUISVILLE, Colo., Dec. 16, 2019 /PRNewswire/ --French Transit, LLC brands MyChelle Dermaceuticalsand CRYSTAL Deodoranttoday announced plans to continue and expand existing partnerships with several key charities and non-profits in 2020. These initiatives are part of an extensive corporate social responsibility program spearheaded by French Transit CEO Martin Floreani.

"It is no longer enough to be 'clean' or 'cruelty-free' in a passive manner," says Floreani. "We also need to be proactive in our stewardship of the planet and its resources. We need to put our time, money and full commitment behind our mission which we define as Clean & Caring." Floreani will be a key panelist during a Natural Products Expo West education seminar in March dedicated to helping other companies define and grow their own corporate social responsibility programs.

"Brands need to embrace cruelty-free research processes, but our company goes beyond by supporting organizations like The Gentle Barn that foster vegan and cruelty-free lifestyles." The Gentle Barn is a non-profit organization dedicated to ending animal cruelty and animal testing that is unfortunately still a reality in the beauty and personal care category. It provides sanctuary for abused and neglected animals and allows inner-city and at-risk children to interact with them, promoting respect and responsibility. MyChelle and CRYSTAL brands supported the Gentle Barn's "Gentle 12 program" for the month of August, and served as title sponsors for the charity's 20th Anniversary Gala in September and have confirmed their renewed commitment for 2020.

MyChelle Dermaceuticals will also continue and expand its support of The Coral Restoration Foundation, the world's largest nonprofit marine-conservation organization dedicatedto restoring our planet's coral reefs to a healthy state. MyChelle was one of the first cosmetic manufacturers to reject marine-toxic ingredients, including oxybenzone, octinoxate, butylparaben, retinyl palmitate and 4-methylbenzylidene camphor. "MyChelle is dedicated not only to creating reef-friendly sun care but also helping organizations such as the Coral Restoration Foundation to preserve and grow reefs," said Floreani. Last July, the company donated 1% of all net proceeds from online sales to the Foundation. This year MyChelle will continue its support of the Coral Restoration Foundation but is also working to be the first brand in its category to incorporate ocean recycled plastic into its packaging.

French Transit is also a recurring sponsor of Breast Cancer Prevention Partners (BCPP), an organization that raises awareness of toxic chemicals used in many personal care products. In 2019, during Breast Cancer Awareness Month, French Transit donated 1% of net sales from their websites to the organization, a commitment that will be repeated in 2020.

"We look forward to building upon our work with these amazing and truly impactful organizations in 2020," said Floreani. "And we are excited and proud to be a leader in a growing community of personal care companies who actively go beyond their claims to foster positive change and real results."

http://www.mychelle.com (PRNewsFoto/MyChelle Dermaceuticals)" alt="Founded in 2000, the Colorado-based skin care company is credited as the first to successfully develop and market natural skin care products using a combination of anti-aging peptides, plant stem cells, and clinically proven dermatological ingredients. Learn more at http://www.mychelle.com (PRNewsFoto/MyChelle Dermaceuticals)">

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SOURCE French Transit

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French Transit Brands to Expand their Corporate Social Responsibility Programs in 2020 - Benzinga

Skin Stem Cells Comments Off on French Transit Brands to Expand their Corporate Social Responsibility Programs in 2020 – Benzinga | December 16th, 2019

However, we may finally have a solution to the First Worlds most enduring problem, baldness, as according to new research, it is certainly possible to create new hair follicles when theyve redeposited themselves to the foot of your shower, through the application of stem-cell technology and 3D printing.

There is a caveat, as the results of the technique are not necessarily long-lasting. When you try to clone hair cells, over time they dedifferentiate and stop producing hair, says Robert Bernstein, a dermatologist in Manhattan who specialises in hair transplantation. For a long time, no one could figure out why. But researchers gradually solved the problem. Over the past few years, they noticed that cells spread out when theyre cultured; the follicular structure essentially melts away. The epiphany was that if you can keep the cells together in their teardrop shape so they continue to signal each other, they continue to grow into hair follicles, Bernstein says.

The next step is to create hair farms, according to an entrepreneur who has started such a thing. Stemson Therapeutics is, per The Atlantic, growing hair from stem cellsnot fetal, but stem cells derived from a persons own skin or bloodand implanting hair follicles rich with dermal papillae into the space around a persons old, shrunken, dormant follicle.

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Science makes baldness optional (if you can afford it) - The Big Smoke Australia

Skin Stem Cells Comments Off on Science makes baldness optional (if you can afford it) – The Big Smoke Australia | December 15th, 2019

For the first time ever, Israeli scientists in Tel Aviv made a 3D printed artificial heart using a patients own cells," proclaims a Washington Post story headlined Researchers create 3D printed heart on April 17 this year.

In the scientific paper published two days earlier, the Israeli scientists describe how they made a bioink out of heart cells and other materials from a patient, and then bioprinted the tissue in the shape of a tiny heart, which was kept alive in a nutrient solution. Their paper makes it clear that this 3D printed heart could not function like a real heart. But the way the research that was projected in media shows how the idea of 3D printed organs is hyped.

Biotechnology has made significant advances, but its still a long way from creating organs that can be transplanted into people. The vasculaturethe network of blood vessels that feeds the organis a challenge.

Stem cell engineering to grow all the cells of an organ in a personalised way to avoid rejection by the recipients immune system is another challenge. And finally, researchers will have to show that a lab organ will work with all the other organs in a human body.

At the same time, the development of 3D bioprinters in the last few years has raised the prospects of making tissues and organs in a more affordable and consistent way because of the speed and precision of the machines. Advances in related fields like nanotechnology and gene editing are also pushing the needle.

These are exciting times, but for startups rushing into this nascent field with huge potential, its as important to be prudent as brave. One way is to go after low hanging fruit instead of the holy grail.

Shift to clinical use

Something like skin is easier to translate into a clinical setting," says Alok Medikepura Anil, director and co-founder of Bengaluru-based 3D bioprinting startup Next Big Innovation Lab (NBIL), which has made human skin in the lab. The skin has good regenerative properties and most of the function of bioprinted skin is to keep infections away, provide nutrition for skin to regenerate and stop the scarring of wounds. Replicating this is easier than replicating the function of a critical organ such as the heart."

This approach contrasts with that of another Bengaluru-based 3D bioprinting startup Pandorum Technologies, founded in 2011 by two researchers at Indian Institute of Science. Pandorum first tried its hand with liver tissue and more recently announced that it had bio-engineered corneal tissue.

Organ tissue for clinical use will require FDA and other approvals. So thats a very expensive proposition," says angel investor Venkat Raju, who took an interest in Pandorum but eventually made a bet on NBIL whose proprietary Innoskin also has non-clinical use in cosmetics testing.

The regulatory environment is evolving. This year, FDA released an RMAT (regenerative medicine and advanced therapy) policy that includes tissue engineering. The FDA wants to fast-track tissue-engineered products if they have a lot of benefits," says Pooja Venkatesh, NBIL co-founder.

Raju feels that startups like NBIL gaining traction and validation could bridge the current gap between academic research and business.

Theres tonnes of research happening across the globe on bioprinting. But universities are struggling to commercialize their research. The fact that NBIL is getting receptive audiences in academia is because they see an opportunity to push their research out."

The Wake Forest Institute of Regenerative Medicine in the US is one of the leading institutions for research in this field. Researchers there are growing tissues for over 40 different areas of the body. They were the first to transplant a lab-grown organ into a 10-year-old patient.

Made-to-order organs

Dr Anthony Atala, who is now the director of the institute, had taken a piece of the boys bladder and grown a new one in the lab over the course of two months. The lab-grown bladder was then transplanted into the patient.

That boy, Luke Massella, went on to become the captain of his school wrestling team. Pretty much I was able to live a normal life after that," Massella, who is now 28, said in a recent interview on BBC.

Stories like that of Massella stoke excitement over futuristic scenarios where you could get made-to-order organs. But researchers admit that there are many unsolved problems in tissue engineering before complex organs like the heart, kidney and liver can be bioprinted. The crash of well-funded San Diego 3D bioprinting startup Organovo, which hit a brick wall in commercializing liver tissue, reminds us to keep the hype in check.

Sumit Chakraberty is a contributing editor with Mint. Write to him at chakraberty@gmail.com.

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Cutting through the hype to get to bioprinted human tissue - Livemint

Skin Stem Cells Comments Off on Cutting through the hype to get to bioprinted human tissue – Livemint | December 15th, 2019

Aspen Neuroscience, a new San Diego biotech company working on stem cell treatment for Parkinsons disease, has come out of stealth mode and raised $6.5 million to pursue clinical testing for its therapy.

Co-founded by well-known stem cell scientist Jeanne Loring, Aspen Neuroscience proposes creating stem cells from modified skin cells of Parkinsons patents via genetic engineering.

The stem cells, which can become any type of cell in the body, then would undergo a process that makes them specialize into dopamine-releasing neurons.

People with Parkinsons lose a large number up to 50 percent at diagnosis of specific brain cells that make the chemical dopamine.

Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems.

Once these modified skin cells have been engineered to specialize in producing dopamine, they can be transplanted into the Parkinsons patient to restore the types of neurons lost to the disease.

The reason we called it Aspen is because l was raised in the Rocky Mountain states, said Loring. When there is a forest fire in the Rockies, the evergreens are wiped out but the aspens are the fist that regenerate after the burn. So it is a metaphor for regeneration.

Aspen still has a long way to go before its proposed therapy would be available to Parkinsons patients. It has been meeting with the U.S. Food and Drug Administration to provide animal trial data and other information in hopes of getting permission to start human clinical trials.

But the company expects the earliest it would get the go-ahead from FDA to start human trials would be 2021.

Loring has been working on the therapy for eight years. She is professor emeritus and founding director of the Center for Regenerative Medicine at the Scripps Research Institute.

Loring co-founded the 20-employee company with Andres Bratt-Leal, a former post-doctoral researcher in Lorings lab at Scripps.

Joining them as Aspens Chief Executive is Dr. Howard Federoff, former vice chancellor for health affairs and chief executive of the University of California Irvine Health System.

Federoff said the company is the only one pursuing the use of Parkinsons patients own cells as part of neuron replacement therapy.

Aspens proprietary approach does not require the use of immuno-suppression drugs, which can be given when transplanted cells come from another person and perhaps limit the effectiveness of the treatment.

Aspens approach is a therapy that is likely to benefit from the fact that your own cells know how to make the best connections with their own target cells in the brain, even in the setting of Parkinsons disease, said Federoff. So when transplanted it is able to set back the clock on Parkinsons.

In addition to Aspens main therapy, it is researching a gene-editing treatment for forms of Parkinsons common in certain families.

Aspens research work up to now has been supported by Summit for Stem Cell, a non-profit on which provides a variety of services for people with Parkinsons disease.

The new seed funding round was led by Domain Associates and Axon Ventures, with additional participation from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32.

Aspens financial backing, combined with its experienced and proven leadership team, positions it well for future success, said Kim Kamdar, a partner at Domain Associates. Domain prides itself on investing in companies that can translate scientific research into innovative medicines and therapies that make a difference in peoples lives. We clearly see Aspen as fitting into that category, as it is the only company using a patients own cells for replacement therapy in Parkinsons disease.

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Aspen Neuroscience gets funding to pursue personalized cell therapy for Parkinsons disease - The San Diego Union-Tribune

Skin Stem Cells Comments Off on Aspen Neuroscience gets funding to pursue personalized cell therapy for Parkinsons disease – The San Diego Union-Tribune | December 14th, 2019

A Campus meeting in November reviewed the arguments for and against donor conception, and the sometimes difficult ethical arguments raised by the prospect of a donor-conceived child. 'Artificial' sperm cells derived from testicular tissue or stem cells may resolve some of those arguments.

The problem is especially acute in cancers diagnosed in prepubertal boys in whom there are no sperm cells available for storage. Their only option for future fatherhood in the face of cancer treatment is adoption or donor sperm. And this, added Goossens, is not an exceptional problem. Incidence rates are around 17 cases per 100,000 population, with leukemia and CNS tumours the most commonly diagnosed. So the usual pathway to fertility preservation in these young cases is for the oncologist to warn of the risk to future fertility from the cancer treatments and refer to the fertility clinic. Biopsy of testicular tissue, of course, must be performed before any radio- or chemotherapy.

Goossens described two experimental techniques, spermatogonial stem cell retrieval and transplantation, and homotopic tissue grafting. The danger in the former procedure is a risk of introducing malignancy, so banked tissue must be free of malignant contamination. Experiments in mouse-to-mouse models have demonstrated spermatogenesis from tissue grafting, and most recently fully functional conception and delivery in a non-human primate (Grady). Similarly, experiments in mouse models with spermatogonial stem cell transplantation have so far proved efficient, with spontaneous pregnancy already possible.

Of course, the objective of this impressive experimental work is not merely a resolution to the question of genetic continuity in couples faced with third-party donation, but the future fertility and long-term quality of life of so many unfortunate young boys. Advances in cancer treatment have led to the increased survival of all children with cancer, and with it a new imperative for the restoration of their fertility. Not all cancer treatments cause complete testicular damage, but around one-third of children having treatment for pediatric cancers will end up infertile. Following the proof-of-concept study which saw the birth of Grady - in which testicular samples removed from prepubertal monkeys was frozen, thawed and regrafted under scrotal skin - the research group declared that their next logical step, with safety and feasibility apparent, is human trials.

1. Fayomi AP, Peters K, Sukhwani M, et al. Autologous grafting of cryopreserved prepubertal rhesus testis produces sperm and offspring. Science 2019; 363: 1314-1319.

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CAMPUS: EGG DONATION - Artificial sperm cells to remove the genetic worries of sperm donation - ESHRE

Skin Stem Cells Comments Off on CAMPUS: EGG DONATION – Artificial sperm cells to remove the genetic worries of sperm donation – ESHRE | December 12th, 2019

The idea of a cell therapy for Parkinsons disease starts out simple: Symptoms of the progressive disease are largely driven by the deaths of dopamine-producing neurons found deep within the brain. With lower levels of the neurotransmitter come the characteristic tremors, rigidity and slow movements.

By replacing those lost nerve cells with new dopamine producers, researchers hope to renew the brains connection to the bodys muscles and improve a persons overall motor function.

But in the brain, everything becomes more complicated. On top of the risk of immune system rejection that comes with any kind of living tissue transplant, its important to make sure the implanted cells function correctly and do not pick up any dangerous genetic mutations as they grow.

How ICON, Lotus, and Bioforum are Improving Study Efficiency with a Modern EDC

CROs are often at the forefront of adopting new technologies to make clinical trials more efficient. Hear how ICON, Lotus Clinical Research, and Bioforum are speeding database builds and automating reporting tasks for data management.

Now, a new company, Aspen Neuroscience, aims to tackle both obstacles at once.

First, the startup hopes to avoid any harmful immune reactions by using a patients own cells as a starting point. Then, Aspen plans to implement a rigorous quality control program employing whole genome sequencing and artificial intelligence to make sure the cells stay in line as theyre processed and readied for the procedure.

And to do it, the San Diego-based company is starting out with $6.5 million in seed money plus an impressive roster of names.

They are led by neurology researcher Howard Federoff, previously vice chancellor for health affairs and CEO of the University of California, Irvine health system as well as the executive dean of medicine at Georgetown University. Hes joined by Aspen co-founder and stem cell scientist Jeanne Loring, founding director and professor emeritus of the Center for Regenerative Medicine at the Scripps Research Institute.

Meanwhile, the seed round was led by Domain Associates and Axon Ventures with additional backing from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32.

Aspen looks to combine its expertise in stem cell biology, genomics and neurology to offer the first autologous cell therapy for Parkinsons diseasewhile others in the space have pursued allogeneic routes, or therapies derived from donors other than the patient.

The process starts with a culture of the patients skin cells, which are then genetically induced to become pluripotent stem cellsor cells capable of differentiating into any other cell type in the body. These are then chemically nudged further to transform into precursor versions of the dopamine-producing neurons, which are typically found in the midbrain and regions responsible for the movement of limbs.

We can say without any equivocation that we can produce the population of cells necessary to transplant, and in a short enough period of time to have a potential beneficial impact on the evolution of the disease, said Federoff, who has also served as chair of the NIHs Recombinant DNA Advisory Committee and helped lead the U.S. Parkinsons Disease Gene Therapy Study Group.

We envisage that this will set back the clock on patients who have Parkinsons, unlike any other therapy that we know of, he told FierceMedTech in an interview.

The number of cells needed would be much smaller compared to other cell therapies and cancer treatments. The healthy human brain contains only about 200,000 dopamine-producing nerve cells, split between its two hemispheres, while patients with Parkinsons disease have lost about 50% or more of those neurons.

Aspen aims to evaluate two doses: one that aims to replace about 60% to 65% of a persons normal cell complement and another larger treatment, Federoff said.

Those smaller doses, as well as starting with a patients donor cells, help make the treatment safer to produce by requiring fewer steps. Each cycle of cell division and multiplication to increase their numbers carries the risk of introducing genetic mutations.

As the cells are grown, they are consistently evaluated with data-driven techniques pioneered by Lorings laboratory. Using whole genome RNA sequencing, Aspen will match the cells up at every stage with a genetic barcode taken from each patient at the start. This will allow them to look for changes, duplications or deletions in the pluripotent stem cell genome.

If the cells harbor mutations that are cancer drivers, we don't want to put those into people, Loring said. The only way is to check the sequencing before we transplant them.

The cells used in the transplant procedure arent fully grown; as neuron progenitors, they mimic the development steps seen in the brain of a growing fetus after theyre placed in the body as they wire themselves up to other neural structures and begin to form new networks of their own.

We anticipate that they will manufacture and release dopamine in a manner that is consistent with synaptic neurotransmission and the process of communicating from cell-to-cell, said Federoff. They will take up dopamine from synapses when it has done its business, bring it back into the cell, and prepare it for another synaptic release.

These are not just dopamine pumps, theyre real neurons, added Loring. They will genuinely replace the cells that have been lost in every way.

Aspen plans to pursue two courses of therapy, for the two major types of Parkinsons disease. Their lead candidate is for idiopathic, or sporadic Parkinsons, while their second is a CRISPR-edited version of the therapy designed to address one of the diseases most common genetic mutations, linked to about 5% of cases.

This would not only aim to restart dopamine production in this orphan indication, but also restore the damaged enzyme GBA, which is seen as an underlying cause. Federoff and Loring expect their sequencing-based quality check system will also help catch any off-target edits linked to the use of CRISPR-Cas9.

The company has yet to secure permission from the FDA to officially launch clinical trials, but the agency has signed off on Aspens plans to prepare a trial-ready cohort of Parkinsons disease patients in the meantime. This would include the initial stages of recruitment and testing, including the selection of patients capable of having their skin cells made into pluripotent stem cells.

After it receives its go-ahead from the FDA, Aspen plans to hit the ground running,enrolling at least 176 participants in a phase 1/2 study that includes a randomized stage to determine clinical benefits.

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By turning stem cells into brain cells, Aspen Neuroscience hopes to rewind the progress of Parkinson's disease - FierceBiotech

Skin Stem Cells Comments Off on By turning stem cells into brain cells, Aspen Neuroscience hopes to rewind the progress of Parkinson’s disease – FierceBiotech | December 12th, 2019

How do you know if your skin is dry? For starters, it feels rough to the touch and, in extreme cases, it may start flaking and you may even feel a tearing sensation.

The good news is that dryskin can be easily avoided, and the most efficient way to hydrate parched skin is with a hydrating sheet mask. All it takes is 15 to 20 minutes and youre on your way to plumped, glowing skin.

These days, hydration sheet masks are loaded with so much extra goodness vitamins, minerals, amino acids,organic acids, plant stem cells andpeptidesthat gointo the formulas that these can also help fight wrinkles, eliminate dark spots and lighten skin tone.

CHARLOTTE TILBURY INSTANT MAGIC FACIAL DRY SHEET MASK, S$76

Yes, this is a dry mask that you can get at Sephora. The magic all comes from the warmth of your skin: The bio-mimetic vector delivery system turns a combo of ingredients (crocus bulb extract, plant stem cells, peptides and vitamin B3) from solid to liquid.

Wear the dry mask by looping the hoops over each ear then simply activate the formula by massaging upwards to move the mask into place. After 15 minutes, take it off then gently tap the remaining essence into the skin. Youll soon notice a glow that is similar to the one you get after a super shiok facial.

But dont throw away the mask just yet the dry formula is engineered for reuse. In fact, you can use it three more times. Just slip it back (the formula side facing inward) into the resealable foil pouch for when you need it next. So really, thats four masks for the price of one.

UTENA PURESA SHEET MASK HYALURONIC ACID 5S, S$12.90

You can pick this up at Watsons. There are five masks to a pack and each one combines the benefits of hyaluronic acid and royal jelly extract, infused with a gel-like essence that works to hydrate skin like a jelly mask. To make the most of it, first smoothen the jelly bits onto the face before placing the sheet over the face.

But theres more: Peek into the packaging and youll see lots of jelly bits left. Apply these remaining bits anywhere else you want to hydrate. We personally like to spread it over the neck, the back of the palms and even the elbows. Yes, it also works as a body hydration gel.

SK II FACIAL TREATMENT MASK, S$127

Love SK IIs Facial Treatment Essence? Then youll love this sheet mask because its drenched with so much Facial Treatment Essence that it feels as if youve dunked your face into a tub of Pitera.

It contains 50 micro-nutrients like vitamins, minerals, amino acids and organic acids to condition skin's natural functions. Twenty minutes is all it takes to rehydrate, clarify complexion and have crystal clear skin.

LA MER THE TREATMENT LOTION HYDRATING MASK, S$45

Each sheet mask is infused with a full ounce of La Mers liquid energy skin hydrator and its equipped with Japanese skin-hugging technology that delivers a concentrated surge of healing hydration directly onto the skin to nourish and soften fine lines.

STARSKIN RED CARPET READY HYDRATING BIO-CELLULOSE SECOND SKIN FACE MASK, S$18

Another mask you can pick up at Sephora is one with a difference. Instead of prepping your face, you prep the mask massage the still-closed sachet to distribute the serum liquid evenly before opening.

The exclusive Bio Cellulose sheet a thin biodegradable microfibre is infused with a delicious cocktail of coconut juice, Amino acids and brown algae that work together to strengthen the skin barrier and promote moisture retention. Dull and dehydrated skin doesnt stand a chance.

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Got dry skin but no time for a facial? These hydration sheet masks are just as good - CNA

Skin Stem Cells Comments Off on Got dry skin but no time for a facial? These hydration sheet masks are just as good – CNA | December 12th, 2019

Materials science company W. L. Gore & Associates Inc. (Newark, DE) has announced a joint venture with the Mayo Clinic (Rochester, MN) to further develop a therapeutic system using stem cells and bio-absorbable scaffolds to treat a condition affecting patients with Crohns disease. Avobis Bio, based in Delaware, will draw on the expertise of scientists and medical professionals from both organizations to build on the encouraging results of an investigational treatment for perianal fistulas.

A debilitating condition that affects patients with Crohns disease, perianal fistulas are painful tunneling wounds connecting the anus to the skin, explained Gore in a press release. Few healing options exist, and patients endure multiple surgeries and ongoing risk of life-threatening complications, said Gore.

"Perianal fistulas are truly life-altering for Crohn's patients, and treatment options have eluded gastroenterologists and surgeons for years," added William Faubion Jr., MD, a Mayo Clinic gastroenterologist who specializes in inflammatory bowel diseases.

The innovative treatment involves harvesting a patients own mesenchymal stem cells, which then are populated on Gore's bioabsorbable polymer scaffold and surgically implanted in the fistula. A phase I clinical trial showed that 76% of patients healed within a year. If this outcome is validated in a larger trial, Gore said that it would dramatically exceed outcomes achieved with existing treatments.

This project is the initial focus of Avobis Bio, which describes its overall mission as an exploration of the use of mesenchymal stem cells combined with enabling bioabsorbable scaffolds that enhance the effectiveness of the cells in stimulating the body to heal.

Delivering a patients mesenchymal stem cells on a synthetic scaffold that biodegrades over time may be a first-of-its-kind medical therapy, noted Joe Carlson, a reporter at the Minneapolis-based Star Tribune reporting on the joint venture. If successful, Avobis Bio may one day offer a variety of tissue and organ-repair therapies combining Mayo's stem cell expertise and Gore's medical materials, he wrote.

Gore is perhaps best known to the public for its Gore-Tex outerwear, but the privately held $3.7 billion engineering and manufacturing firm sells products in an array of industries, including a line of medical devices designed to repair nonnatural holes in body organs, added Carlson. Mayo has used Gore-made devices for many years.

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Gore, Mayo Clinic team up to deliver breakthrough medical therapies - Plastics Today

Skin Stem Cells Comments Off on Gore, Mayo Clinic team up to deliver breakthrough medical therapies – Plastics Today | December 12th, 2019

This article highlights some of the most recent drug target discoveries that could be used to develop and design a treatment for pancreatic cancer.

Scientists investigating pancreatic cancer have identified new targets which, with further research, could be the basis for developing future therapies. Listed below are five of the most recent target discoveries, in order of their journal publication dates, with the newest first.

Scientists at the Queen Mary University of London, UK and Zhengzhou University, China have developed a personalised vaccine system that may be able to delay the onset of pancreatic cancer.

Cells taken from mice, mutated chemically into pancreatic cancer cells and then infected with Adenovirus (AdV) as a prime or Vaccinia virus (VV) as a boost, create a vaccine product. The virus kills the cancerous cells in such a way that their antigens are released and are therefore able to prime the immune system to prevent pancreatic cancer returning.

Injection of the virus-infected cells into mice destined to develop pancreatic cancer doubled their survival rate, compared to their unvaccinated counterparts. The vaccine also delayed the onset of the condition in these mice.

Using cells from the recipient of the vaccine enables the immune system to respond to the exact antigens seen in tumour cells of the individual, resulting in a vaccine regime tailored to them.

Through this international collaboration, we have made progress towards the development of a prophylactic cancer vaccine against pancreatic cancer, said Professor Yaohe Wang, leader of the study, from Queen Mary University of London and the Sino-British Research Centre at Zhengzhou University in China.

Researchers at Sanford Burnham Prebys Medical Discovery Institute in the US have identified that a combination of two anti-cancer compounds, already approved for use to treat other cancers, shrank pancreatic tumours in mice.

Our study identifies a potential treatment combination that can immediately be tested against these aggressive tumours. We are already meeting with oncologists at Oregon Health & Science University, US to discuss how to advance this discovery into clinical evaluation, explained Dr Zeev Ronai, a professor in Sanford Burnham Prebys Tumor Initiation and Maintenance Program, also senior author of the study.

Scientists used L-asparaginase to starve pancreatic tumours of asparagine, an amino acid required by cells for protein synthesis. However, the tumour cells did not die, instead switching on a stress response pathway whereby they could produce asparagine themselves. Scientists then used an MEK inhibitor to block the stress response pathway, causing the pancreatic tumour to shrink.

L-asparaginase is already US Food and Drug Administration (FDA) approved to treat leukaemias and similarly the MEK inhibitor is approved for the treatment of solid tumours, including melanoma skin cancer.

This research lays the basis for the inhibition of pancreatic tumour growth by a combined synergistic attack based on asparagine restriction and MAPK signalling inhibition, says Dr Eytan Ruppin, chief of the Cancer Data Science Library at the National Cancer Institute (NCI) and co-author of the study.

Scientists from the Max Planck Institute for Biology of Ageing, Germany have identified that YME1L, a protease in the membrane of mitochondria, is activated when a cell uses glycolysis to produce energy anaerobically.

scientists were able to reduce tumour growth by switching off the glycolysis signalling pathway in the mitochondria

Cells adapt to oxygen deficiency by switching their energy supply to glycolysis, which ferments sugar without oxygen. This switch is often necessary in old age, as the cells in the body become poorly supplied with oxygen and nutrients.

Cancer cells can also face this problem; prior to angiogenesis, tumours are poorly perfused and so the tissue is deprived of oxygen. Oxidative stress in tumours drives the switch-on of multiple pathways. This includes the glycolysis pathway that alters the behaviour of the mitochondria to provide tumour cells with energy despite being starved of oxygen.

Scientists found that the YME1L protease is activated during the conversion to glycolysis. YME1L appears altered and breaks down various proteins in the organelles, preventing the formation of new mitochondria and causing the remaining organelles to change their metabolism. This process eventually stops as YME1L begins to degrade itself at high activity.

Researchers examined cancer cells originating from patients with pancreatic tumours and were able to reduce tumour growth by switching off the glycolysis signalling pathway in the mitochondria, with reproducible results both in the petri dish and in pancreatic tumours in mice.

There is currently no treatment available for pancreatic cancer. I believe that this protease can be a very interesting therapeutic target because we have seen that the signalling pathway is also active in human patients with pancreatic cancer, explained Thomas Langer, the Max Planck Director, continuing: However, there are no known substances that have an effect on this protease.

Researchers at the Crick Institute have identified cancer stem cells as a driver of pancreatic cancer growth. These cells can metastasise and differentiate into different tumour types to continue the spread of cancer.

Cancer stem cells appear at all stages of cancer growth so being able to identify where they are present could be vital in both targeting cancer and developing new treatments, according to the researchers. Analysis of gene expression in the cancer stem cells identified a protein, CD9, is present on tumour surfaces during development and when it is more established. This protein could therefore be used as a marker to help locate these cells.

A further development of the study established that this protein is not just a marker of cancer stem cells, but also promotes their malignant behaviour. By altering the amount of CD9 in tumour cells in mice, researchers found that reduced levels of this protein caused smaller tumours to form and increasing levels of CD9 created more aggressive cells able to form large tumours quickly.

These cells are vital to pancreatic cancer and if even just a few of them survive chemotherapy, the cancer is able to bounce back. We need to find effective ways to remove these cells and so stop them from fuelling cancer growth. However, we need more experiments to validate the importance of CD9 in human pancreatic cancer, says Victoria Wang, lead author and member of the Adult Stem Cell Laboratory at the Crick Institute.

A look into cancer stem cell metabolism also revealed CD9 increases the rate tumour cells take up glutamine, an amino acid which helps provide energy for cancer growth.

Now we know this protein is both linked to cancer stem cells and helps cancer growth, this could guide the development of new treatments that are targeted at the protein and so cut off the supply of glutamine to cancer stem cells, effectively starving the cancer, says Axel Behrens, corresponding author and group leader in the Adult Stem Cell Laboratory at the Crick Institute.

Scientists at Tel Aviv University, Israel have found that PJ34, a small molecule, causes human pancreatic cancer cells to self-destruct. The researchers tested PJ34 on xenografts (transplants) of human pancreatic tumours in mice.

this mechanism also exists in other types of cancer and therefore the treatment could be valuable for use on those resistant to current therapies

The mice were treated with a molecule called PJ34, which is permeable in the cell membrane but affects human cancer cells exclusively. This molecule causes an anomaly during the duplication of human cancer cells, provoking their rapid cell death. Thus, cell multiplication itself resulted in cell death in the treated cancer cells, explains Professor Malca Cohen-Armon, project lead at Tel Aviv Universitys Sackler Faculty of Medicine.

The treatment consisted of daily PJ34 injections for 14 days and four weeks later there was a relative drop of 90 percent in the number of cancer cells within the tumours of the mice. Cohen-Armon also noted there were no adverse side-effects observed in the mice.

This mechanism similarly exists in other types of cancer and therefore the treatment could be valuable for use on those resistant to current therapies. The molecule PJ34 is being tested in pre-clinical trials according to FDA regulations before clinical trials begin.

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Five recent drug target discoveries for pancreatic cancer - Drug Target Review

Skin Stem Cells Comments Off on Five recent drug target discoveries for pancreatic cancer – Drug Target Review | December 12th, 2019

Im hairythats just fact, and its something Ive always been aware of. Ill never forget being in summer camp as a kid, wearing shorts every day and becoming very aware that my legs were much hairier than those of the girls around me. Now, Im no longer ashamed of being hairyIve actually come to embrace it. But in the past two decades, Ive gotten very acquainted with epilators, including laser hair removal.

I got my first laser hair removal treatment two years ago. It seemed like everyone was getting it done, but as a black woman, I knew I wasnt everyone. For brown skin, laser hair removal isnt a spur-of-the-moment decision, a process that should be approached through a Groupon or the local nail salon (yes, some nail salons actually perform laser hair removal). For us, the process can be a bit more expensive and tedious, and should be approached with the utmost caution.

Why? Well, lasers target pigment, and due to the high content of melanin in our skin, risks of discoloration and hyper-pigmentation are astronomically higher. So thats why, according to Chris Karavolas, owner of Romeo And Juliette Laser Hair Removal, darker skin requires a completely different laser all together, and operators with much more experience. Darker skin complexions need to be careful because not all centers have the right lasers for dark skin, and even if they do they do, many do not have enough experience in treating dark skin, he says.

When it comes to those specialized lasers, there are two options. Its important to treat with an Nd:YAG laser, such as the Candela GentleYAG or GentleMax Pro, says Anne Chapas, M.D., medical director of Union Square Laser Dermatology in New York City. The wavelength of a YAG laser goes deeper into the skin than a diode laser and is less absorbed by the surrounding skin pigment, so it more successfully treats the stem cells of the hair follicle. Additionally, more treatments may be required than for fairer skin: Expect to receive at least six sessions, Chapas says.

Knowing all these things, I went into laser hair removal with cautious optimism. I chose to treat my Brazilian areayears of improper hair removal had left me with ingrown and severe discoloration, to the point that my wax lady refused to continue treating me because my skin had gotten so irritated. I was sure that I wanted to continue being hair-free down there, so I decided to get laser hair removal on my vagina area. I was nervous heading into my first appointment, so I made sure to ask the aesthetician at Romeo & Juliette in-depth questions about her experience with dark skin, as well as requesting to see photos of previous clients and inquiring about a patch test. Then, it was showtime.

I like to think that I have a pretty high pain tolerance. However, laser removal certainly made me question that belief. I wont lie and say that it didnt hurt. It did. It felt like being snapped, hard, by rubber bands, but it was quick, and the results I saw were almost immediate. I was advised to shave right before the treatment, in order to give the laser immediate access to the hair follicle, and because, well, nobody likes the smell of burning hair. After about 10 minutes, I was sent on my way and advised to come back in another six weeks. Laser hair removal requires a strict schedule and works in conjunction with the hair cycle for optimal results. Every appointment thereafter was just as easy, and now, completely through all six of my sessions, my hair is almost completely gone, with the occasional stubble appearing here and there.

So how do you know if your skin will need one of these specialized lasers? Different ethnicitiesregardless of skin tonereact differently to lasers. Your skin may appear to be a 3 or 4 on the Fitzpatrick scale, but if youre, say, Latin or Asian, it could react to the laser as a 6 would, Dr. Chapas says. So be sure that youre only going to facilities that have operators trained in dermatology, and are skilled enough to differentiate your skin tone.

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What its like to get laser hair removal as a black woman - Yahoo Lifestyle

Skin Stem Cells Comments Off on What its like to get laser hair removal as a black woman – Yahoo Lifestyle | December 12th, 2019

Researchers in China are laying claim to the first ever pig-monkey chimeras to be born in what they hope will be a breakthrough for biomedicine, not just fuel for your nightmares.

The worlds first monkey-pig hybrids have been born in a Chinese lab amid scientific attempts to grow human organs inside animals. The incredible experiment saw two chimera piglets born with DNA from both pigs and cynomolgus monkeys. However, the experiment was short-lived as the two piglets both died within a week.

According to the researchers, the baby piglets had genetic material from cynomolgus monkeys in their heart, liver, spleen, lung and skin.

However, the success rate was significantly low as only 10 piglets were born from more than 4,000 implanted in sows.

From those, only two were chimeras in a result that has been deemed discouraging by other leading stem cell scientists.

Lead researcher Tang Hai at the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing said: This is the first report of full-term pig-monkey chimeras.

Ms Hai confirmed that it was unclear why the piglets died, but the non-chimeric pigs died as well.

The Chinese research team suspect that the deaths could have been due to the IVF process instead than the chimerism

According to research study, stem cells from macaque monkeys were grown in a lab and then injected into pig embryos five days after fertilisation.

Ms Hai said that the next step in their research was to create healthy animals with a higher proportion of monkey cells before creating pigs in which one organ is composed almost entirely of primate cells.

The ultimate research goal is to grow human organs inside live animals as a way to resolve the crisis of organ transplantation.

University of California stem cell biologist Paul Knoepfler said: Given the extremely low chimeric efficiency and the deaths of all the animals, I see this as fairly discouraging.

Reaction to the study has prompted many social media users to question if this is a step too far.

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Researchers Manage to Create Pig-Monkey Chimeras for the First Time - Tdnews

Skin Stem Cells Comments Off on Researchers Manage to Create Pig-Monkey Chimeras for the First Time – Tdnews | December 10th, 2019

Theglobal market for cell harvestingshould grow from $885 million in 2018 to reach $1.5 billion by 2023 at a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023.

Report Scope:

The scope of the report encompasses the major types of cell harvesting that have been used and the cell harvesting technologies that are being developed by industry, government agencies and nonprofits. It analyzes current market status, examines drivers on future markets and presents forecasts of growth over the next five years.

The report provides a summary of the market, including a market snapshot and profiles of key players in the cell harvesting market. It provides an exhaustive segmentation analysis of the market with in-depth information about each segment. The overview section of the report provides a description of market trends and market dynamics, including drivers, restraints and opportunities. it provides information about market developments and future trends that can be useful for organizations, including wholesalers and exporters. It provides market positionings of key players using yardsticks of revenue, product portfolio, and recent activities. It further includes strategies adopted by emerging market players with strategic recommendations for new market entrants. Readers will also find historical and current market sizes and a discussion of the markets future potential. The report will help market players and new entrants make informed decisions about the production and exports of goods and services.

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Report Includes:

41 data tables and 22 additional tables Description of segments and dynamics of the cell harvesting market Analyses of global market trends with data from 2017, 2018, and projections of compound annual growth rates (CAGRs) through 2023 Characterization and quantification of market potential for cell harvesting by type of harvesting, procedure, end user, component/equipment and region A brief study and intact information about the market development, and future trends that can be useful for the organizations involved in Elaboration on the influence of government regulations, current technology, and the economic factors that will shape the future marketplace Key patents analysis and new product developments in cell harvesting market Detailed profiles of major companies of the industry, including Becton, Dickinson and Co., Corning, Inc., Fluidigm Corp., General Electric Co., Perkinelmer, Inc., and Thermo Fisher Scientific, Inc.

Summary

Stem cells are unspecialized cells that have the ability to divide indefinitely and produce specialized cells. The appropriate physiological and experimental conditions provided to the unspecialized cells give rise to certain specialized cells, including nerve cells, heart muscle cells and blood cells. Stem cells can divide and renew themselves over long periods of time. These cells are extensively found in multicellular organisms, wherein mammals, there are two types of stem cells embryonic stem cells and adult stemcells. Embryonic stem cells are derived from a human embryo four or five days old that is in the blastocyst phase of development. Adult stem cells grow after the development of the embryo and are found in tissues such as bone marrow, brain, blood vessels, blood, skin, skeletal muscles and liver. Stemcell culture is the process of harvesting the exosomes and molecules released by the stem cells for the development of therapeuticsfor chronic diseases such as cancer and diabetes. The process is widely used in biomedical applications such as therapy, diagnosis and biological drug production. The global cell harvesting market is likely to witness a growth rate of REDACTED during the forecast period of 2018-2023.The value of global cell harvesting market was REDACTED in 2017 and is projected to reach REDACTED by 2023. Market growth is attributed to factors such as increasing R&D spending in cell-based research,the introduction of 3D cell culture technology, increasing government funding, and the growing prevalence of chronic diseases such as cancer and diabetes.

The growing incidence and prevalence of cancer is seen as one of the major factors contributing to the growth of the global cell harvesting market. According to the World Health Organization (WHO), cancer is the second-leading cause of mortality globally and was responsible for an estimated 9.6 million deaths in 2018. Therefore, there is an increasing need for effective cancer treatment solutions globally. Cell harvesting is the preferred method used in cancer cell-related studies including cancer cell databases (cancer cell lines), and other analyses and drug discovery in a microenvironment. The rising prevalence of such chronic diseases has led governments to provide R&D funding to research institutes and biotechnology companies to develop advanced therapeutics. Various 3D cell culture technologies have been developed by researchers and biotechnology companies such as Lonza Group and Thermo Fischer Scientific for research applications such as cancer drug discovery. The application of cell culture in cancer research is leading to more predictive models for research, drug discovery and regenerative medicine applications.

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Platelet-rich plasma (PRP) therapy, a new biotechnology solution that has a heightened interest among researchers in tissue engineering and cell-based therapies, has various applications in the treatment of tissue healing in tendinopathy, osteoarthritis and muscle injury. It has been conventionally employed in orthopedics, maxillofacial surgery, periodontal therapy and sports medicines. PRP therapy can be used in the treatment of fat grafting, acne scars, and hair regrowth.

Major factors driving market growth include increasing healthcare costs and the high rate of adoption for modern medicines in emerging economies such as China and India. It has been estimated that India will witness a CAGR of REDACTED in the cell harvesting market during the forecast period. The active participation of foreign pharmaceutical companies has tapped the Indian healthcare sector with a series of partnerships and mergers and acquisitions, which in turn is positively impacting the growth of the market in this region. Consistent development and clinical trials for stem cell therapies, plus contribution from the government and private sectors through investments and cohesive reimbursement policies in the development of cancer biomarkers, is further fueling market growth. InSweden, a research team at Lund University has developed a device to collect fluid and harvest stem mesenchymal stem cells (MSCs). The device is developed with 3D-printed bio-inert plastics which, when used by doctors, can result in the safe extraction of fluids (medical waste) from the patients body. The liquid is then passed through a gauze filter for purifying thoroughly and MSCs are separated from the fluid by centrifugation and are grown in culture.

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Cell Harvesting Market a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023 - Crypto News Byte

Skin Stem Cells Comments Off on Cell Harvesting Market a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023 – Crypto News Byte | December 10th, 2019

WALTHAM, Mass., Dec. 10, 2019 /PRNewswire/ --Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that it plans to commence a Phase 2 expansion cohort based on encouraging clinical activity and a well-tolerated safety profile observed to date in the ongoing Phase 1 dose escalation trial of SNDX-6352 in patients with chronic graft versus host disease (cGVHD). SNDX-6352 is the Company's anti-CSF-1R monoclonal antibody.

The ongoing Phase 1, open-label, modified 3+3 dose escalation trial is designed to evaluate the safety and preliminary efficacy of SNDX-6352 in up to 30 patients with cGVHD who have received at least two prior lines of therapy. As of a November 25, 2019 data cutoff date, a total of five patients, all of whom received prior treatment with ibrutinib, steroids, and a calcineurin inhibitor, have been enrolled across three dose cohorts: one patient was treated at 0.15 mg/kg every two weeks (Q2W, Cohort 1), one is receiving a dose of 0.5 mg/kg Q2W (Cohort 2), and three patients are receiving 1.0 mg/kg Q2W (Cohort 3).

Responses have been observed in all evaluable patients as of the data cutoff date, with no dose limiting toxicities (DLTs) reported. Among the three patients dosed in Cohort 3 (1.0 mg/kg Q2W), one patient recently cleared the DLT period and has not yet been evaluated for efficacy, two patients experienced a partial response, and all three patients remain on therapy. The patient in Cohort 2 experienced a partial response and is currently in their ninth month of treatment with SNDX-6352 following prior treatment with ibrutinib and both Jakafi (ruxolitinib) and KD025, two agents currently being investigated for the treatment of cGVHD. The first patient (Cohort 1) achieved a partial response but discontinued in their third cycle due to elevated LFTs attributed to progression in their liver cGVHD. Cohort 4, which will explore a 3.0 mg/kg Q2W dose, is now open for enrollment.

"The initial results from our Phase 1 trial underscore the potential of SNDX-6352 to serve as an effective therapy for patients with cGVHD who are lacking alternative options," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We had not anticipated commenting on data from this initial trial until the second half of 2020, so it is quite encouraging to see the early signs of activity in patients with this difficult to treat disease. Based on these results, we have decided to advance into a Phase 2 expansion cohort to evaluate additional patients at the 1.0 mg/kg dose while we continue the dose escalation to 3.0 mg/kg. We continue to expect to present the Phase 1 trial results in the second half of 2020.

"Published preclinical data have demonstrated that CSF-1R blockade can prevent and treat disease in animal models of cGVHD1," said Peter Ordentlich, Ph.D., Chief Scientific Officer and Co-founder of Syndax."The initial data from our trial provide the first clinical evidence that targeting CSF-1R dependent macrophages may benefit patients with cGVHD."

To date, SNDX-6352 has been safe and well-tolerated, with no DLTs observed. Dose escalation is ongoing in the Phase 1 portion of the trial. The Phase 2 expansion cohort is expected to enroll up to 22 patients to further characterize the safety and efficacy at an initial dosing schedule of 1.0 mg/kg of SNDX-6352 administered every two weeks.

About Chronic Graft Versus Host Disease

Chronic graft versus host disease (cGVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT) which can last for years. cGVHD is estimated to develop in approximately 40% of transplant recipients, and affect approximately 14,000 patients in the US. 2-4 cGVHD typically manifests across multiple organ systems, with the skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue.

About SNDX-6352

SNDX-6352 is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages and the development of cutaneous and pulmonary chronic graft versus host disease (cGVHD), as well as to lead to the depletion of cells known as Tumor Associated Macrophages, or TAMS. SNDX-6352 is currently being evaluated in a Phase 1 multiple ascending dose clinical trial in cGVHD, and a Phase 1 multiple ascending dose clinical trial as monotherapy and in combination with Infinzi (durvalumab) in solid tumors. SNDX-6352 has the potential to treat a variety of solid tumor and immune-related diseases.

About Syndax Pharmaceuticals, Inc.

Syndax Pharmaceuticalsis a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. The Company's lead product candidate, entinostat, a once-weekly, oral, small molecule, class I HDAC inhibitor, is being tested in a Phase 3 combination trial with exemestane for treatment of advanced HR+, HER2- breast cancer and has been evaluated in combination with several approved PD-1/PD-(L)1 antagonists. The Company's pipeline also includes SNDX-6352, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor, and SNDX-5613, a highly selective inhibitor of the MeninMLL binding interaction. For more information, please visitwww.syndax.comor follow the Company on TwitterandLinkedIn.

Syndax's Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend," "believe" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials and the reporting of clinical data for Syndax's product candidates, and the potential use of our product candidates to treat various cancer indications. Many factors may cause differences between current expectations and actual results including unexpected safety or efficacy data observed during preclinical or clinical trials, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of Syndax's collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

References

1. Alexander, KA. et al. J Clin Invest. 2014;124(10):42664280.

2.Kantar GVHD Expert Interviews N=8 interviews

3. SmartAnalyst 2017 SmartImmunology Insights chronic GVHD report.

4. Bachier, CR. et al. ASH annual meeting 2019; abstract #2109 Epidemiology and Real-World Treatment of Chronic Graft-Versus-Host Disease Post Allogeneic Hematopoietic Cell Transplantation: A U.S. Claims Analysis.

Syndax Contacts

Investor ContactMelissa ForstArgot Partnersmelissa@argotpartners.com212.600.1902

Media ContactCraig HeitGCI Healthcraig.Heit@gcihealth.com347.451.4733

SNDX-G

SOURCE Syndax Pharmaceuticals, Inc.

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Syndax Pharmaceuticals Announces Plans to Commence Phase 2 Expansion Cohort of SNDX-6352 for the Treatment of Chronic Graft Versus Host Disease -...

Skin Stem Cells Comments Off on Syndax Pharmaceuticals Announces Plans to Commence Phase 2 Expansion Cohort of SNDX-6352 for the Treatment of Chronic Graft Versus Host Disease -… | December 10th, 2019

Pigs engineered to have a small amount of monkey cells have been brought to full term and were even born alive, surviving for a few days after birth. Although the piglets died, it is claimed the experiment - performed in China - marks a major milestone for the future of lab-grown organs.

"This is the first report of full-term pig-monkey chimeras," Tang Hai of the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing told New Scientist.

The research is part of an ongoing effort to develop animals - whether they are sheep or pigs - that can grow human organs we could then harvest for transplants, a process called xenogeneic organogenesis.

Research has been done on both pig and sheep embryos with transplanted human stem cells; in both cases, the embryos continued to develop until the experiment was deliberately terminated.

That's because, due to ethical concerns, these chimeras - organisms that incorporate the genetic material of another species - cannot be cultivated or studied in the later stages of embryonic development. Some scientists worry that some of the human stem cellscould end upin other parts of the animal or even in its brain, with unintended consequences.

For that reason, in this experiment the team used stem cells from crab-eating macaques (Macaca fascicularis). These were imbued with fluorescent proteins so that they would glow under fluorescent light, and derived to produce fluorescing embryonic cells.

These cells were then injected into over 4,000 five-day-old pig embryos fertilised using IVF; the modified pig embryos were subsequently implanted into sows.

This fiddly and painstaking work produced just 10 piglets that made it to full term and were born alive. And only two of these were chimeric, with between one in 1,000 and one in 10,000 functional monkey cells to pig cells.

The monkey cells had migrated to the heart, liver, lungs, spleen and skin of the piglet hosts, but were not found in other organs, such as testes and ovaries, due to the low rate of chimerism, the researchers said.

Sadly, before a week was out, the piglets died - not just the two chimeras, but the other eight normal piglets, too. Because all the pigs died, Hai told New Scientist, the cause of death likely had less to do with chimerism, and more to do with IVF - a procedure that is notoriously tricky in pigs.

The low chimerism rate is also somewhat discouraging. However, the researchers remain optimistic. Although the birth rate was low, and the pigs didn't survive, the team now has a wealth of data they can apply to future experiments.

The scientists are planning to try again, increasing the chimeric cell ratio. And they believe their data may help other scientists working in the field.

"Here, we have used monkey cells to explore the potential of reconstructing chimeric human organs in a large animal model," they wrote in their paper.

"We believe this work will facilitate the development of xenogeneic organogenesis by providing a better understanding of the processes of xenogeneic recognition, fate determination, and the proliferation and differentiation of primate stem cells during porcine development.

"The findings could pave the way toward overcoming the obstacles in the re-engineering of heterogeneous organs and achieve the ultimate goal of human organ reconstruction in a large animal."

The research has been published in Protein & Cell.

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Pig-Monkey Chimeras Have Been Brought to Term For The First Time - ScienceAlert

Skin Stem Cells Comments Off on Pig-Monkey Chimeras Have Been Brought to Term For The First Time – ScienceAlert | December 9th, 2019

ORLANDO, Fla.--(BUSINESS WIRE)--Amnion of Florida, a leading provider of alternative medicine utilizing cryopreserved placental cell allograft and advanced bioactive facial rejuvenation, is pleased to announce their vendor choice to round out regenerative anti-aging therapies.

Amnion announces a partnership with Merakris Therapeutics, LLC to advance the development of Merakris topical bioactive anti-aging hydrogel technology. Christopher Broderick, President, and Founder of Merakris Therapeutics stated, dedication to science-based outcomes is our primary focus, thus were delighted to be selected based upon our scientific approach to youth maintenance and rejuvenation technologies.

Amnion is focused on attracting women and men seeking affordable non-surgical options for youth maintenance via cell activated procedures, hair restoration, joint repair, dermal rejuvenation, and anti-aging treatments.

Our team of experienced medical professionals and aestheticians at Amnion are excited to utilize the Merakris Therapeutics product suite, including medical-grade, sterile filtered amniotic fluid serums and hydrogels at our newest Spa in Sanford, FL, said Eusebio Coterillo, President of Amnion.

In a constantly changing field, Amnion of Florida, under the guidance of the on-site medical staff, provides the highest level of quality products and procedures in cosmetic medicine. They offer cutting edge treatments that are proven by research, the use FDA cleared or registered products, and are widely published and peer endorsed.

More about Amnion of Florida

Amnion of Florida, based in Central Florida, is a leading provider of alternative medicine using cryopreserved placental cell transplants or allografts, processed from donated cellular birth tissue, which are natural alternatives to autologous regenerative medicine products. The primary function of our allogeneic regenerative treatments is to promote soft tissue joint/skin repair and regeneration mediated by growth factors and cells naturally found in placental tissue. These treatments have shown safety and efficacy in treating a variety of ailments including osteoarthritis, chronic ulcerative wounds, joint pain, skin rejuvenation, hair restoration, urinary incontinence, and ED. Learn more http://www.amnion.us.

More about Merakris Therapeutics, LLC

Merakris Therapeutics, based in Research Triangle Park, North Carolina, is focused on researching, developing, and marketing regenerative healthcare products. Merakris is pioneering commercially scalable biotherapeutic technologies derived from stem cells that have various clinical applications. Our vision is to improve global patient care and outcomes through the pioneering and innovation of acellular regenerative biotechnologies. Learn more at http://www.merakris.com.

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Top Florida Medical Spa, Amnion of Florida, Partners With Merakris Therapeutics to Advance Their Non-Surgical Treatment Options - Business Wire

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SEATTLE--(BUSINESS WIRE)--Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, presented Phase 1 data yesterday from the healthy volunteer study of ALPN-101, a first-in-class dual CD28/ICOS antagonist, and details on its upcoming Phase 1/2 BALANCE study of ALPN-101 in steroid-resistant or steroid-refractory acute graft-versus-host disease (GVHD) at the 61st American Society of Hematology Annual Meeting (ASH) in Orlando, FL.

Jan Hillson, MD, Senior Vice President of Clinical Development at Alpine, presented An Open Label Study of ALPN-101, a First-in-Class Dual CD28/ICOS Antagonist, in Subjects with Steroid-Resistant or Steroid-Refractory Acute Graft Versus Host Disease (BALANCE) as part of the oral session, Chemical Biology and Experimental Therapeutics: Novel Compounds and Mechanisms of Action.

Highlights included:

Despite decades of intense research, GVHD remains a major cause of morbidity and mortality after hematopoietic stem cell transplantation, commented Sophie Paczesny, MD, PhD, Professor of Immunology and Pediatrics at Indiana University School of Medicine and lead of the Biomarkers Stem Cell Transplantation Program and one of Alpine's research collaborators. Current therapies are associated with significant toxicities or are simply insufficient to control the disease. CD28 and ICOS appear to be key pathways in the pathogenesis of GVHD, and the presented data with ALPN-101 appear uniquely strong. I look forward to the BALANCE study, which may demonstrate the therapeutic potential of ALPN-101.

About Graft Versus Host Disease (GVHD)

Graft versus host disease (GVHD) is the most common life-threatening complication of a hematopoietic cell transplant. It occurs when donor cells see recipient cells as foreign and attack them. Acute GVHD typically occurs within the early weeks and months after transplant, usually involving the skin, liver, and gastrointestinal tract. GVHD patients remain at risk of organ system damage and increased mortality due to the disease and to high dose glucocorticoids.

About ALPN-101

ALPN-101 is a novel Fc fusion protein of a human inducible T cell costimulator ligand (ICOSL) variant immunoglobulin domain (vIgD), and a first-in-class therapeutic designed to inhibit simultaneously the CD28 and ICOS inflammation pathways. CD28 and ICOS are closely-related costimulatory molecules with partially overlapping roles in T cell activation likely playing a role in multiple autoimmune and inflammatory diseases. In preclinical models of graft versus host disease, inflammatory arthritis, connective tissue disease, and multiple sclerosis, ALPN-101 demonstrates efficacy superior to agents blocking the CD28 CD80/86 or ICOS - ICOSL pathways alone.

About Alpine Immune Sciences, Inc.

Alpine Immune Sciences, Inc. is committed to leading a new wave of immune therapeutics, creating potentially powerful multifunctional immunotherapies to improve patients lives via unique protein engineering technologies. Alpine has two lead programs. The first, ALPN-101 for autoimmune/inflammatory diseases, is a selective dual T cell costimulation blocker engineered to reduce pathogenic T and B cell immune responses by blocking ICOS and CD28. ALPN-101 has recently completed enrollment in a Phase 1 healthy volunteer trial. The second, ALPN-202 for cancer, is a conditional CD28 costimulator and dual checkpoint inhibitor. Alpine is backed by world-class research and development capabilities, a highly-productive scientific platform, and a proven management team. For more information, visit http://www.alpineimmunesciences.com.

Forward-Looking Statements

This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are not based on historical fact and include statements regarding our platform technology and potential therapies, the timing of and results from clinical trials and pre-clinical development activities, clinical and regulatory objectives and the timing thereof, expectations regarding the sufficiency of cash to fund operations, the potential efficacy, safety profile, future development plans, addressable market, regulatory success, and commercial potential of our product candidates, the timing of our public presentations and potential publication of future clinical data, the efficacy of our clinical trial designs, expectations regarding our ongoing collaborations, and our ability to successfully develop and achieve milestones in our development programs. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions and include words such as may, will, should, would, expect, plan, intend, and other similar expressions, among others. These forward-looking statements are based on current assumptions that involve risks, uncertainties, and other factors that may cause actual results, events, or developments to be materially different from those expressed or implied by such forward-looking statements. These risks and uncertainties, many of which are beyond our control, include, but are not limited to: clinical trials may not demonstrate safety and efficacy of any of our product candidates; our ongoing discovery and pre-clinical efforts may not yield additional product candidates; our discovery-stage and pre-clinical programs may not advance into the clinic or result in approved products; any of our product candidates may fail in development, may not receive required regulatory approvals, or may be delayed to a point where they are not commercially viable; we may not achieve additional milestones in our proprietary or partnered programs; the impact of competition; adverse conditions in the general domestic and global economic markets; as well as the other risks identified in our filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof and we undertake no obligation to update forward-looking statements, and readers are cautioned not to place undue reliance on such forward-looking statements.

Secreted Immunomodulatory Proteins, SIP, Transmembrane Immunomodulatory Protein, TIP, Variant Ig Domain, vIgD and the Alpine logo are registered trademarks or trademarks of Alpine Immune Sciences, Inc. in various jurisdictions.

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Alpine Immune Sciences Presents ALPN-101 Phase 1 Healthy Volunteer Study Data and Details of Upcoming Phase I/II BALANCE GVHD Study at the 61st...

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Beijing: In a world first, researchers have been able to produce pig-monkey hybrids in a Chinese laboratory, a media report said. Two piglets - having monkey tissues in their hearts, liver and skin - were born in the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing but died within a week, the Mirror reported.

The announcement to this effect was made by Tang Hai. The experiment comes in the wake of Spanish scientist Juan Carlos Izpisua Belmonte's attempt to create pig-human hybrids two years ago. Tang and his team injected genetically modified monkey cells into more than 4,000 pig embryos before these were implanted into sows, the New Scientist magazine reported.

Only two of the 10 piglets thus born were hybrids - with tissues in the heart, liver, spleen, lung and skin, partly consisting of monkey cells. Experts suspected that the failure has to do with the IVF process.

Critics have now warned that the scientific development will create "disturbing" dilemmas over the ethics of human-animal hybrids. Doctor Angel Raya, director of the Barcelona Regenerative Medicine Centre, told the Spanish daily El Pais: "What happens if the stem cells escape and form human neurons in the brain of the animal? Would it have consciousness? And what happens if these stem cells turn into sperm cells?''

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Meet the world's first pig-monkey hybrid - Free Press Journal

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Our lives have been transformed by the information age. But what's coming next is likely to be more profound, call it the genetic information age. We have mapped the human genome and in just the last few years we have learned to read and write dna like software. And you're about to see a few breakthroughs-in-waiting that would transform human health. For a preview of this revolution in evolution we met George Church, a world leading geneticist, whose own DNA harbors many eccentricities and a few genes for genius.

We found George Church in here.Cory Smith: Most of these are frozen George. Little bits of George that we have edited all in different tubes.

Church threw himself into his work, literally. His DNA is in many of the experiments in his lab at Harvard Medical School. The fully assembled George Church is 6'5" and 65. He helped pioneer mapping the human genome and editing DNA. Today, his lab is working to make humans immune to all viruses, eliminate genetic diseases, and reverse the effects of time.

Scott Pelley: One of the things your lab is working on is reversing aging.

George Church: That's right.

Scott Pelley: How is that possible?

George Church: Reversing aging is one of these things that is easy to dismiss to say either we don't need it or is impossible or both.

Scott Pelley: Oh, we need it.

George Church: Okay. We need it. That's good. We can agree on that. Well, aging reversal is something that's been proven about eight different ways in animals where you can get, you know, faster reaction times or, you know, cognitive or repair of damaged tissues.

Scott Pelley: Proven eight different ways. Why isn't this available?

George Church: It is available to mice.

In lucky mice, Church's lab added multiple genes that improved heart and kidney function and levels of blood sugar. Now he's trying it in spaniels.

Scott Pelley: So is this gene editing to achieve age reversal?

George Church: This is adding genes. So, it's not really editing genes. It's, the gene function is going down, and so we're boosting it back up by putting in extra copies of the genes.

Scott Pelley: What's the time horizon on age reversal in humans?

George Church: That's in clinical trials right now in dogs. And so, that veterinary product might be a couple years away and then that takes another ten years to get through the human clinical trials.

Human trials of a personal kind made George Church an unlikely candidate to alter human evolution. Growing up in Florida, Church was dyslexic, with attention deficit, and frequently knocked out by narcolepsy.

Scott Pelley: What was it that made you imagine that you could be a scientist?

George Church: The thing that got me hooked was probably the New York World's Fair in 1964. I thought this is the way we should all be living. When I went back to Florida, I said, "I've been robbed," you know? "Where is it all?" So, I said, "Well, if they're not going to provide it, then I'm gonna provide it for myself."

With work and repetition, he beat his disabilities and developed a genius for crystallography, a daunting technique that renders 3D images of molecules through X-rays and math. But in graduate school at Duke, at the age of 20, his mania for the basic structures of life didn't leave time for the basic structure of life.

Scott Pelley: You were homeless for a time.

George Church: Yeah. Briefly.

Scott Pelley: Six months.

George Church: Six months.

Scott Pelley: And where were you sleeping when you were homeless?

George Church: Well, yeah. I wasn't sleeping that much. I was mostly working. I'm narcoleptic. So, I fall asleep sitting up anyway.

His devotion to crystallography was his undoing at Duke.

George Church: I was extremely excited about the research I was doing. And so, I would put in 100-plus hours a week on research and then pretty much didn't do anything else.

Scott Pelley: Not go to class.

George Church: I wouldn't go to class. Yeah.

Duke kicked him out with this letter wishing him well in a field other than biology. But, it turned out, Harvard needed a crystallographer. George Church has been here nearly 40 years. He employs around 100 scientists, about half-and-half men and women.

Scott Pelley: Who do you hire?

George Church: I hire people that are self-selecting, they see our beacon from a distance away. There are a lot of people that are a little, you know, might be considered a little odd. "Neuroatypicals," some of us are called.

Scott Pelley: "Neuroatypical?"

George Church: Right.

Scott Pelley: Unusual brains?

George Church: Right, yeah.

Parastoo Khoshakhlagh: One thing about George that is very significant is that he sees what you can't even see in yourself.

Parastoo Khoshakhlagh and Alex Ng are among the "neuroatypicals." They're engineering human organ tissue.

Cory Smith: I think he tries to promote no fear of failure. The only fear is not to try at all.

Cory Smith's project sped up DNA editing from altering three genes at a time to 13,000 at a time. Eriona Hysolli went to Siberia with Church to extract DNA from the bones of wooly mammoths. She's editing the genes into elephant DNA to bring the mammoth back from extinction.

Eriona Hysolli: We are laying the foundations, perhaps, of de-extinction projects to come.

Scott Pelley: De-extinction.

Eriona Hysolli: Yes.

Scott Pelley: I'm not sure that's a word in the dictionary yet.

Eriona Hysolli: Well, if it isn't, it should be.

Scott Pelley: You know there are people watching this interview who think that is playing God.

George Church: Well, it's playing engineer. I mean, humans have been playing engineer since the dawn of time.

Scott Pelley: The point is, some people believe that you're mucking about in things that shouldn't be disturbed.

George Church: I completely agree that we need to be very cautious. And the more powerful, or the more rapidly-moving the technology, the more cautious we need to be, the bigger the conversation involving lots of different disciplines, religion, ethics, government, art, and so forth. And to see what it's unintended consequences might be.

Church anticipates consequences with a full time ethicist in the lab and he spends a good deal of time thinking about genetic equity. Believing that genetic technology must be available to all, not just those who can afford it.

We saw one of those technologies in the hands of Alex Ng and Parastoo Khoshakhlagh. They showed us what they call "mini-brains," tiny dots with millions of cells each. They've proven that cells from a patient can be grown into any organ tissue, in a matter of days, so drugs can be tested on that patient's unique genome.

Scott Pelley: You said that you got these cells from George's skin? How does that work?

Alex Ng: We have a way to reprogram essentially, skin cells, back into a stem cell state. And we have technologies where now we can differentiate them into tissue such as brain tissue

Scott Pelley: So you went from George's skin cells, turned those into stem cells, and turned those into brain cells.

Alex Ng: Exactly. Exactly.

Scott Pelley: Simple as that.

Organs grown from a patient's own cells would eliminate the problem of rejection. Their goal is to prove the concept by growing full sized organs from Church's DNA.

George Church: It's considered more ethical for students to do experiments on their boss than vice versa and it's good to do it on me rather than some stranger because I'm as up to speed as you can be on the on the risks and the benefits. I'm properly consented. And I'm unlikely to change my mind.

Alex Ng: We have a joke in the lab, I mean, at some point, soon probably, we're going to have more of his cells outside of his body than he has himself.

Church's DNA is also used in experiments designed to make humans immune to all viruses.

George Church: We have a strategy by which we can make any cell or any organism resistant to all viruses by changing the genetic code. So if you change that code enough you now get something that is resistant to all viruses including viruses you never characterized before.

Scott Pelley: Because the viruses don't recognize it anymore?

George Church: They expect a certain code provided by the host that they replicate in. the virus would have to change so many parts of its DNA or RNA so that it can't change them all at once. So, it's not only dead. But it can't mutate to a new place where it could survive in a new host.

Yes, he's talking about the cure for the common cold and the end of waiting for organ transplants. It's long been known that pig organs could function in humans. Pig heart valves are routinely transplanted already. But pig viruses have kept surgeons from transplanting whole organs. Church's lab altered pig DNA and knocked out 62 pig viruses.

Scott Pelley: What organs might be transplanted from a pig to a human?

George Church: Heart, lung, kidney, liver, intestines, various parts of the eye, skin. All these things

Scott Pelley: What's the time horizon on transplanting pig organs into human beings?

George Church: you know, two to five years to get into clinical trials. And then again it could take ten years to get through the clinical trials.

Church is a role model for the next generation. He has co-founded more than 35 startups. Recently, investors put $100 million into the pig organ work. Another Church startup is a dating app that compares DNA and screens out matches that would result in a child with an inherited disease.

George Church: You wouldn't find out who you're not compatible with. You'll just find out who you are compatible with.

Scott Pelley: You're suggesting that if everyone has their genome sequenced and the correct matches are made, that all of these diseases could be eliminated?

George Church: Right. It's 7,000 diseases. It's about 5% of the population. It's about a trillion dollars a year, worldwide.

Church sees one of his own genetic differences as an advantage. Narcolepsy lulls him several times a day. But he wakes, still in the conversation, often, discovering inspiration in his twilight zone.

Scott Pelley: If somebody had sequenced your genome some years ago, you might not have made the grade in some way.

George Church: I mean, that's true. I would hope that society sees the benefit of diversity not just ancestral diversity, but in our abilities. There's no perfect person.

Despite imperfection, Church has co-authored 527 scientific papers and holds more than 50 patents. Proof that great minds do not think alike.

The best science can tell, it was about 4 billion years ago that self-replicating molecules set off the spark of biology. Now, humans hold the tools of evolution, but George Church remains in awe of the original mystery: how chemistry became life.

Scott Pelley: Is the most amazing thing about life, then, that it happened at all?

George Church: It is amazing in our current state of ignorance. We don't even know if it ever happened ever in the rest of the universe. it's awe-inspiring to know that it either happened billions of times, or it never happened. Both of those are mind boggling. It's amazing that you can have such complex structures that make copies of themselves. But it's very hard to do that with machines that we've built. So, we're engineers. But we're rather poor engineers compared to the pseudo engineering that is biological evolution.

Produced by Henry Schuster. Associate producer, Rachael Morehouse. Broadcast associate, Ian Flickinger.

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Harvard geneticist George Church's goal: to protect humans from viruses, genetic diseases, and aging - 60 Minutes - CBS News

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