MRC MBU (@mrc-mbu.bsky.social)

Clinically translatable mitochondrial gene therapy in muscle using tandem mtZFN architecture | EMBO Molecular Medicine imageimageEfficient mitochondrial gene therapy is achieved with a novel tandem zinc finger nuclease (mtZFN) architecture delivered via adeno-associated viral (AAV) vectors, significantly reducing mutation loads in muscle tissue and rescuing ... https://www.embopress.org/doi/full/10.1038/s44321-025-00231-5