GeneticsMolecular Therapy — Methods & Clinical DevelopmentPeer-reviewed
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…·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.