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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主要な著者:	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
フォーマット:	Artigo
言語:	Inglês
出版事項:	Elsevier 2023-09-01
シリーズ:	Molecular Therapy: Methods & Clinical Development
主題:	gene therapy hemophilia B adeno-associated virus vectors vector recoverability intra-hepatic vascular administration
オンライン･アクセス:	http://www.sciencedirect.com/science/article/pii/S2329050123001304
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