Huge congrats to Pavel Nash on publishing his PhD work! 🎉 We showcase a safer mtDNA gene therapy using a novel mtZFN architecture compatible with a single AAV. Thanks to the entire team!
@mrc-mbu.bsky.social
@mbu-postdocs.bsky.social #MitochondrialDisease #mtDNA
www.embopress.org/doi/full/10....
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 ...
