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Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy

The first generation of adeno-associated virus (AAV) vectors composed of the naturally occurring capsids and genomes, although effective in some instances, are unlikely to be optimal for gene therapy in humans. The use of the first generation of two different AAV serotype vectors (AAV9 and AAVrh74)...

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Detalles Bibliográficos
Autores principales: Shoti, Jakob, Qing, Keyun, Keeler, Geoffrey D., Duan, Dongsheng, Byrne, Barry J., Srivastava, Arun
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10690633/
https://www.ncbi.nlm.nih.gov/pubmed/38046199
http://dx.doi.org/10.1016/j.omtm.2023.101147