A potential therapy for musculardystrophy: Using mRNA delivery to improve muscle strength
It may be only a tiny change in the genome, but this small difference can have fatal consequences: Muscular dystrophies are almost always caused by a single faulty gene. As different as the mutations are in this group of approximately 50 disorders, they all ultimately lead to a very similar outcome.
"We have for several years been pursuing the idea of taking muscle stem cells from diseased patients, using CRISPR-Cas9 to correct the faulty genes, and then injecting the treated cells back into the muscles so that they can proliferate and form new muscle tissue," explains Dr. Helena Escobar, a postdoctoral researcher in Spuler's lab and co-author of the current paper.
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