I'll raise this idea a second time - (this GT room's elephant?) | SRPT Message Board Posts

Sarepta Therapeutics Inc.

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Msg  13792 of 13816  at  6/7/2019 3:11:25 PM  by

schmiggins


I'll raise this idea a second time - (this GT room's elephant?)

This time a SGMO lover posts the thought again today:

"Dystrophin replacement or skipping approaches by gene editing could be limited by natural muscle
turnover/regeneration, which may dilute the therapeutic effect; gene
editing of satellite stem cells could prevent this therapeutic dilution"

The elephant in the room. New muscle cells on growing boys won't have the new gene is the thought. Seems to be unavoidably true. How fast that new muscle growth makes a noticeable difference I have no idea. Maybe it's a very slow moving phenomena. I'd guess the older the boy is when he gets the shot the longer he'd benefit because his muscles are fully developed and not replacing themselves very fast - compared to, say, a 4 year old. I'd like to see Mendel take on that question and give his thinking.

As for the satellite stem cells being a target for gene replacement as a way to bypass this phenomena, I'm not certain and I could go back to the most recent article we posted here from Gerbach's lab, but I seem to recall he said he was going after those satellite stem cells. Anyone remember that?


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Msg # Subject Author Recs Date Posted
13793 Re: I'll raise this idea a second time - (this GT room's elephant?) schmiggins 0 6/7/2019 3:22:23 PM


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