Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines.
Wei SC, Cantor AJ, 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 WI, Shahi PK, Chan J, Boivin P, Otoupal P, Pattnaik BR, Gamm DM, Saha K, Gowen BG, Haak-Frendscho M, Janatpour MJ, Silverman AP.
Wei SC, et al.
bioRxiv [Preprint]. 2024 Dec 30:2024.12.30.630799. doi: 10.1101/2024.12.30.630799.
bioRxiv. 2024.
PMID: 39803585
Free PMC article.
Preprint.