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Comparative in vivo characterization of newly discovered myotropic adeno-associated vectors

Abstract Background Adeno-associated virus (AAV)-based gene therapy is a promising strategy to treat muscle diseases. However, this strategy is currently confronted with challenges, including a lack of transduction efficiency across the entire muscular system and toxicity resulting from off-target t...

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Autores principales: Jacqueline Ji, Elise Lefebvre, Jocelyn Laporte
Formato: Artigo
Lenguaje:Inglês
Publicado: BMC 2024-05-01
Colección:Skeletal Muscle
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Acceso en línea:https://doi.org/10.1186/s13395-024-00341-7
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