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New #MolTherapyAdv paper: Preclinical efficacy of a gene therapy for CHKB-mediated muscular dystrophy. #Myoblue tinyurl.com/3vjanc7f
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Congenital megaconial muscular dystrophy (OMIM #602541) is an autosomal recessive disease caused by rare variants in the CHKB gene. McMaster and colleagues carried out a dose-ranging study of rAAV9 ge...
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Preclinical efficacy of a gene therapy for CHKB-mediated muscular dystrophy
Matthew Alexander, PhD