Nemekhbayar Baatartsogt

This paper introduces a cuffed guide RNA (cgRNA) that enables miRNA activity-dependent genome editing in human and mouse cells.

This paper develops engineered FVIII variants for hemophilia A gene therapy, showing improved secretion, coagulation activity, and efficacy at lower vector doses in mice and macaques.

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

This paper develops a single AAV vector system for liver-targeted gene knock-in therapy in mouse models of inherited liver diseases, achieving efficient therapeutic gene insertion without germline transmission.

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.