I strongly recommend this video. At least it lifted me out of the gloom I had got from the Prosensa news.
45 minute description on how to create the disease in a mouse model and then how to fix it too. This lady from Stanford is very close.
tulika, helen blau told about the new mouse model over 2 years ago...this was discussed here as well..
Here is the discission on ppmd from 2 years ago on Helen Blau's work.
My understanding is that Helen is saying that creating dystrophin is not the end state. That will work only in the initial an young age children before the heavy demand on the muscle stem cells causes the Telomeres to shorten.
First paper in 2010 proved that skeletal muscles are impacted by Telomeres shortening and the paper in 2013 is saying that even cardiac function detoriation has a direct connect with Telomeres shortening
Either ways, where new or old, the description and proof given is very simple and logical. Helen is saying that making dystrophin is not the end game, we need to replenish the MuSC. Having dystrophin will only reduce the dependency for MuSC replenishment.