Efficient Gene Transduction in Pigs and Macaques with the Engineered AAV Vector AAV.GT5 for Hemophilia B Gene Therapy
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, , Tsukasa Ohmori
This study evaluates the efficacy and immunogenicity of the AAV.GT5 vector for hemophilia B gene therapy in pigs and macaques, finding improved transduction and reduced immunogenicity compared to AAV-Spark100.
Molecular Therapy — Methods & Clinical Development · Aug 22, 2023Peer-reviewedGenetics