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and J.S.C.) and grant RRG 715234 from the Muscular Dystrophy Association (to J.S.C.). Author Contributions Conceptualization, N.E.B., S.D.H., and J.S.C.; Methodology, N.E.B., H.T., S.D.H., and J.S.C.; Investigation, N.E.B. muscle types. These results suggest that the high rate of necrosis and regeneration in skeletal muscles, compared with the relative stability of dystrophic cardiomyocytes, caused the rapid loss of edited genomes. Consequently, stable dystrophin expression in DMD skeletal muscles will require either highly efficient gene editing or the use of cotreatments that decrease skeletal muscle Gilteritinib hemifumarate degeneration. mice display a uniquely accelerated pace of turnover during an early crisis period that occurs between 3 and 7?weeks postnatally.19 This provides an opportunity for conducting stringent tests Mouse monoclonal to AURKA of AAV-mediated dystrophin gene correction and of the resulting gene-edited dystrophins during…