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, Rina J. Mepani, Kory Melton, Ashil Bans, Prachi Khekare, Suhani Gupta, Jonathan Wang, Craig Soares, Radwan Kiwan, J.L. Lee, Shannon McCawley, Vihasi Jani, Weng In Leong, , 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.
PLoS ONE · Jun 24, 2025Peer-reviewedPharmacology & Toxicology