Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease

Vector production scale-up is a major barrier in systemic adeno-associated virus (AAV) gene therapy. Many scalable manufacturing methods have been developed. However, the potency of the vectors generated by these methods has rarely been compared with vectors made by transient transfection (TT), the...

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Detalles Bibliográficos
Autores principales: Hakim, Chady H., Clément, Nathalie, Wasala, Lakmini P., Yang, Hsiao T., Yue, Yongping, Zhang, Keqing, Kodippili, Kasun, Adamson-Small, Laura, Pan, Xiufang, Schneider, Joel S., Yang, N. Nora, Chamberlain, Jeffrey S., Byrne, Barry J., Duan, Dongsheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7403893/
https://www.ncbi.nlm.nih.gov/pubmed/32775499
http://dx.doi.org/10.1016/j.omtm.2020.07.004

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