Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy

Dystrophin: The protein product of the duchenne muscular dystrophy locus

Eric P. Hoffman, Robert H. Brown, Louis M. Kunkel · Cell · 1987 · 4.6K citations

Protein Function, Biochemistry, Skeletal Muscle +8

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Amit C. Nathwani, Edward G. D. Tuddenham, Savita Rangarajan et al. · New England Journal of Medicine · 2011 · 1.8K citations · Full text

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

Carmela Zincarelli, Stephen Soltys, Giuseppe Rengo et al. · Molecular Therapy · 2008 · 1.4K citations · Full text

Functional improvement of dystrophic muscle by myostatin blockade

Sasha Bogdanovich, Thomas Krag, Elisabeth R. Barton et al. · Nature · 2002 · 819 citations

Muscle Function, Skeletal Muscle, Physiology +2

Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8

Katsuya Inagaki, Sally Fuess, Theresa A. Storm et al. · Molecular Therapy · 2006 · 585 citations · Full text