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Hi, I'm new. Well, not really, but I have been reading quietly the last three years.
Sorry for hijacking your thread, might or might not do it again. :-B

The good thing is that by now there seems to be more therapies researched than I can keep track of. Some that would be considered cures. The bad thing is I've started to notice what seems to be unspoken challenges with more of them. 

I was actually reading about another approach, (SU9516), had a concern and came here to see if anyone were discussing it. And I found I have a similar question with the article you're linking to, as to the article I was reading. So, I hope you don't mind if I ask for opinions here. 

From the article about REV-ERB:

A simplified version of muscle cells' life-cycle looks like this: muscle stem cells produce myoblasts that will either reproduce (proliferate) or form muscle tissue (differentiate). Successful regeneration of skeletal muscle after traumatic injury depends on the replenishment of muscle fibers through elevated myoblast proliferation and differentiation.

Scientists were fascinated to see that REV-ERB appears to play different roles for different stages of muscle tissue development. A decline in expression of REV-ERB precedes myoblast differentiation. Conversely, an increase in REV-ERB expression is involved in the regulation of mitochondrial and metabolic function in fully differentiated skeletal muscle.

No doubt this is useful knowledge, and it seems to me this research might lead to better understanding/manual control of muscle regeneration, possibly optimizing the process. But as I first was reading up on research, I seem to have read about a cancelled study. There were promising results at first with alleviating the symptoms, but then the effect dropped and it was in fact making things worse. What I learned from reading this - or so I think I did - is that one of the challenges with DMD is stem cell depletion. 

I can't help but wonder if simulating muscle regeneration (as in the other article I read) would burn through the stem cell reserves faster, and similarly that manipulating myoblasts to form muscle tissue (differentiate) would do the same? Considering these would either reproduce (proliferate) or differentiate. 

I guess I should include an excerpt that caused my initial concern from the article I was reading about cancer therapy against MD. Despite my somewhat pessimistic outlook, every bit of research helps understand.  

Rather than trying to fix or replace the broken gene, SU9516 ramps up the muscle repair process, helping reinforce muscle structure.


The screen revealed that SU9516 raised integrin production and promoted the formation of muscle cells and fibers from DMD muscle stem cells


“Integrin stabilizes muscle structure, and helps stimulate muscle repair and regeneration,” Burkin said. “If we can artificially increase its production with drugs, we think it can help protect muscle cells from damage.”

Cancer therapy SU9516 repurposed for DMD


There is a researcher Dr. Helen Blau at Standford University that has shown stem cell depletion is a major problem and working on ways to reverse it. 

However, if exon-skipping, utrophin modulation or both are started early stem cell depletion can be slowed or normalized.  For older patients stem cell therapy will be needed. 

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