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PAM-flexible Cas9-mediated base editing of a hemophilia B mutation in induced pluripotent stem cells by Takafumi Hiramoto, Yuji Kashiwakura, Morisada Hayakawa, Nemekhbayar Baatartsogt, Nobuhiko Kamoshita, Tomoyuki Abe, Hiroshi Inaba, Hiroshi Nishimasu, Hideki Uosaki, Yutaka Hanazono, Osamu Nureki, Tsukasa Ohmori
Published 2023-04-01
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Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy by 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
Published 2023-09-01
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