Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
Spencer C. Wei, Aaron J. Cantor, Jack Walleshauser…·Adam P. Silverman
This study evaluates subretinal delivery of Cas9 ribonucleoproteins in mice and pigs, showing variable editing efficiency and highlighting species-specific challenges for therapeutic translation.