Hyperactive PIGGYBAC Transposons for Sustained and Robust Liver-targeted Gene Therapy. Vrije Universiteit Brussel
The development of robust non-viral vectors could facilitate clinical gene therapy applications and may overcome some of the immune complications of viral vectors. Nevertheless, most non-viral gene deliver approaches typically yield only transient and/or low gene expression. To address these caveats, we have explored piggyBac transposons to correct hemophilia B by liver-directed factor IX (FIX) gene therapy in hemophilic mice. To achieve this, ...