Muscular dystrophies are approximately 50 devastating, untreatable monogenic diseases leading to progressive muscle degeneration and atrophy. Gene correction of transplantable cells using CRISPR/Cas9-based tools is a realistic scenario for autologous cell replacement therapies to restore organ function in many genetic disorders. However, muscle stem cells have so far lagged behind due to the absence of methods to isolate and propagate them and their susceptibility to extensive ex vivo manipulations. Here, the researchers show that their mRNA-based delivery method results in up to >90% efficient genome editing in human muscle stem cells from many donors regardless of age and gender and without any enrichment steps. The researchers show that cells edited with mRNA fully retain their myogenic marker signature, proliferation capacity, and functional attributes. Moreover, they achieved a highly efficient repair of a muscular dystrophy-causing SGCA mutation in a single selection-free step. In summary, this work establishes mRNA-mediated delivery of CRISPR/Cas9-based tools as a promising and universal approach for taking gene-edited muscle stem cells into clinical application to treat muscle disease.
mRNA-mediated delivery of gene editing tools to human primary muscle stem cells
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