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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...

詳細記述

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書誌詳細
出版年:Mol Ther Methods Clin Dev
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Gene & Cell Therapy 2020
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC7403893/
https://ncbi.nlm.nih.gov/pubmed/32775499
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.07.004
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