Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy

Gene therapy using adeno-associated virus (AAV)-based vectors has become a realistic therapeutic option for hemophilia. We examined the potential of a novel engineered liver-tropic AAV3B-based vector, AAV.GT5, for hemophilia B gene therapy. In vitro transduction with AAV.GT5 in human hepatocytes was...

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Detalles Bibliográficos
Autores principales: Kashiwakura, Yuji, Endo, Kazuhiro, Ugajin, Atsushi, Kikuchi, Tomohiro, Hishikawa, Shuji, Nakamura, Hitoyasu, Katakai, Yuko, Baatartsogt, Nemekhbayar, Hiramoto, Takafumi, Hayakawa, Morisada, Kamoshita, Nobuhiko, Yamazaki, Shoji, Kume, Akihiro, Mori, Harushi, Sata, Naohiro, Sakata, Yoichi, Muramatsu, Shin-ichi, Ohmori, Tsukasa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10491835/
https://www.ncbi.nlm.nih.gov/pubmed/37693948
http://dx.doi.org/10.1016/j.omtm.2023.08.016

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10491835/
https://www.ncbi.nlm.nih.gov/pubmed/37693948
http://dx.doi.org/10.1016/j.omtm.2023.08.016

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