Takafumi Hiramoto

This study uses base editing to create a universal FIX variant for hemophilia B treatment, increasing FIX activity in cell and mouse models.

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.

This study shows that re-administering different AAV serotypes can overcome neutralizing antibody barriers in mice, enabling successful liver transduction with switched serotypes.