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Systemic delivery of AAV5, AAV8, and AAV9 packaging a C5-12-microdystrophin-FLAG expression cassette in non-human primates

Safely achieving therapeutic expression levels with adeno-associated virus (AAV) gene therapy is a significant challenge for treating the large muscle mass in humans. Non-human primates (NHPs) provide a more accurate assessment of the feasibility of achieving an effective and safe dose than rodents....

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Principais autores: Mengping Liu, Erica Cook, Yanshan Dai, Erich Ehlert, Francois du Plessis, Jacek Lubelski, Bogdan G. Sleczka, Petia Shipkova, Zhuyin Li, Joshua Gamse, David Gordon, Leonard P. Adam, Paul C. Levesque, Glen B. Banks
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2025-03-01
coleção:Molecular Therapy: Methods & Clinical Development
Assuntos:
adeno-associated virus
promoter
systemic delivery
non-human primate
dystrophin
AAV
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2329050125000063
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