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Molecular Therapy. Methods & Clinical Development|September 25, 2023
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophyMichael R Emami, Alejandro Espinoza, Courtney S Young, et al.
Hypertension (Dallas, Tex. : 1979)|February 18, 2009
Onset of experimental severe cardiac fibrosis is mediated by overexpression of Angiotensin-converting enzyme 2Rachel Masson, Stuart A Nicklin, Margaret Anne Craig, et al.
Human Gene Therapy|January 25, 2023
Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical TrialsJeffrey S Chamberlain, Melissa Robb, Serge Braun, et al.
Nature|October 28, 2008
Sarcolemma-localized nNOS is required to maintain activity after mild exerciseYvonne M Kobayashi, Erik P Rader, Robert W Crawford, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 20, 2007
Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscleMaja Z Salva, Charis L Himeda, Phillip Wl Tai, et al.
Proceedings of the National Academy of Sciences of the United States of America|September 6, 2018
Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophyPeggy P Ho, Lauren J Lahey, Foteini Mourkioti, et al.
Neuromuscular Disorders : NMD|December 27, 2022
Clinical potential of microdystrophin as a surrogate endpointJessica F Boehler, Kristy J Brown, Margaret Beatka, et al.
Cell|April 19, 2023
Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene deliverySajedah M Hindi, Michael J Petrany, Elena Greenfeld, et al.
Molecular Therapy. Methods & Clinical Development|September 22, 2017
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular DystrophyChady H Hakim, Nalinda B Wasala, Xiufang Pan, et al.
Molecular Therapy. Methods & Clinical Development|August 11, 2020
Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle DiseaseChady H Hakim, Nathalie Clément, Lakmini P Wasala, et al.
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