Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy
Rachael A. Potter,
Danielle A. Griffin,
Kristin N. Heller
et al.
Abstract:Robust expression of shortened, functional dystrophin provided impetus to develop adeno-associated virus (AAV)–based constructs for clinical application. Because several cassettes are being tested in clinical trials, this study compared efficacy of the following four shortened dystrophin-promoter combinations with implications for outcomes in clinical trials: MHCK7 or MCK promoter with a shortened dystrophin transgene containing the N-terminus and spectrin repeats R1, R2, R3 and R24 (rAAVrh74.MHCK7.micro-dystr… Show more
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