Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines

Gamm, MD, PhD, D., and K. Saha, PhD. Evaluation of Subretinally Delivered Cas9 Ribonucleoproteins in Murine and Porcine Animal Models Highlights Key Considerations for Therapeutic Translation of Genetic Medicines.
Wei, S. C., Cantor, A. J., Walleshauser, J., Mepani, R., Melton, K., Bans, A., Khekare, P., Gupta, S., Wang, J., Soares, C., Kiwan, R., Lee, J., McCawley, S., Jani, V., Leong, W. I., Shahi, P. K., Chan, J., Boivin, P., Otoupal, P., Pattnaik, B. R., … Silverman, A. P. (2025). Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines. PloS one, 20(6), e0317387. https://doi.org/10.1371/journal.pone.0317387