iPS Cell Therapy – Parent Project Muscular Dystrophy

By Dr. Matthew Watson

iPS Cells and Therapeutic Applications for Duchenne

We are currently in the optimization/validation phase of pre-clinical development.

This research is being done in the lab of Dr. Rita Perlingeiro at the University of Minnesota, in partnership with the University of Minnesota Center for Translational Medicine and the Molecular and Cellular Therapeutics Facility. This work is currently funded by the Department of Defense (DoD).

Induced pluripotent stem cells (iPS) are adult cells that have been reprogrammed to an embryonic stem cell-like state.There has been tremendous excitement for the therapeutic potential of iPS cells in treating genetic diseases. Our current research builds on our successful proof-of-principle studies for Duchenne performed with mouse wild-type and dystrophic iPS cells as well as control (healthy) human iPS cells. These studies demonstrate equivalent functional myogenic engraftment to that observed with their embryonic counterparts following their transplantation into dystrophic mice.

Our goal now is to apply this technology to clinical grade GMP-compliant iPS cells, and generate a cell product, iPS-derived myogenic progenitors, that can be delivered to muscular dystrophy patients.

Optimization of methodology, characterization of cell product, scalability with GMP-compliant method, followed by safety and efficacy studies. Once these have been achieved, we will be ready to move into a clinical trial.

2-3 years (it depends largely on how much funding we have available to conduct these studies).

University of Minnesota

In the first phase, adults with confirmed diagnosis of Duchenne (> 18 years old).

You can learn more about this research at the website for Dr. Perlingeiros lab: http://www.med.umn.edu/lhi/research/PerlingeiroLab/index.htm

http://www.ClinicalTrials.gov will post all clinical trials once they are actively recruiting patients.

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iPS Cell Therapy - Parent Project Muscular Dystrophy

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