Increasing stem cell survival in Duchenne therapy

One approach to improving muscle strength in Duchenne is to replace unhealthy muscle cells that lack dystrophin with donor stem cells that have normal levels of dystrophin. These cells might come from a relative unaffected by Duchenne or even a person with Duchenne’s own cell, after dystrophin is restored though a technique like gene therapy.


Although we’ve heard much more in recent years about drug approaches to therapy, work continues to better understand how to use stem cells to restore muscle tissue. In a new article from Purdue, Dr. Shihuan Kuang describes how stem cell therapy would work for Duchenne and his discovery that mimicking the environment in the muscle is the best way to grow healthy muscle stem cells in culture dishes.


Sharon Hesterlee, Ph.D.
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Comment by Dawn Steele on June 28, 2014 at 4:41pm

Thank you.  I was just curious since I've read drug research being completed for Duchenne.  My understanding my son's mutation isn't part of the current research being completed with the exon crossing.  So, was wondering what the stem cell research is finding.  I'm sure this won't be anything testing on the public but was wondering anyways.

Comment by Sharon Hesterlee on June 28, 2014 at 4:21pm
Yes, we are funding Dr. Rita Perlingeiro at the university of Minnesota to develop induced pluripotent stem cells for duchenne. She extracts fibroblasts, converts them to muscle stem cells and then puts dystrophin back into the cells before reintroducing them. For these experiments she's putting corrected human cells back into dystrophic mice, but the idea is that you would be able to do the same for someone with duchenne.
Comment by Dawn Steele on June 28, 2014 at 3:30pm

Is there any more research going on in the stem cell testing to cure Duchenne?

Comment by adi hazan on September 14, 2012 at 3:34pm

יפה שיהיה להם בהצלחה.

Comment by Sharon Hesterlee on August 29, 2012 at 9:41pm

Hi Traci:

I think we just haven't figured out how to get stem cells to work efficiently--it's not that it's a bad idea, but much like gene therapy, there has been a lot of "going back to the drawing board."  I would tend to agree that the ultimate solution for Duchenne is likely to involve some combination of gene and cell therapies, but in the meantime some other therapeutic approaches are closer to approval.  The reason we wanted to post this article is so that people would remember that stem cells are still part of the mix as a possible therapy.  We are reviewing a stem cell project for funding now that would use "induced pluripotent stem cells"--that is cells from a living person like skin cells that are re-programmed to become stem cells--no controversial sources.

Comment by Traci Strafuss on August 29, 2012 at 9:22pm

I hardily have a response.  I believe very strongly that stem cells are such an important part of the "cure"  or part of the "treatment".  Why does there seem to be so much scepticism when it comes tp stem cells/


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