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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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Автори: Yuji Kashiwakura, Kazuhiro Endo, Atsushi Ugajin, Tomohiro Kikuchi, Shuji Hishikawa, Hitoyasu Nakamura, Yuko Katakai, Nemekhbayar Baatartsogt, Takafumi Hiramoto, Morisada Hayakawa, Nobuhiko Kamoshita, Shoji Yamazaki, Akihiro Kume, Harushi Mori, Naohiro Sata, Yoichi Sakata, Shin-ichi Muramatsu, Tsukasa Ohmori
Формат: Artigo
Мова:Inglês
Опубліковано: Elsevier 2023-09-01
Серія:Molecular Therapy: Methods & Clinical Development
Предмети:
gene therapy
hemophilia B
adeno-associated virus vectors
vector recoverability
intra-hepatic vascular administration
Онлайн доступ:http://www.sciencedirect.com/science/article/pii/S2329050123001304
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