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

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

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Genetic medicines, including CRISPR/Cas technologies, extend tremendous promise for addressing unmet medical need in inherited retinal disorders and other indications; however, there remain challenges for the development of therapeutics. Herein, we evaluate genome editing by engineered Cas9 ribonucl...

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Bibliographic Details
Main Authors: Spencer C Wei, Aaron J Cantor, Jack Walleshauser, Rina Mepani, Kory Melton, Ashil Bans, Prachi Khekare, Suhani Gupta, Jonathan Wang, Craig Soares, Radwan Kiwan, Jieun Lee, Shannon McCawley, Vihasi Jani, Weng In Leong, Pawan K Shahi, Jean Chan, Pierre Boivin, Peter Otoupal, Bikash R Pattnaik, David M Gamm, Krishanu Saha, Benjamin G Gowen, Mary Haak-Frendscho, Mary J Janatpour, Adam P Silverman
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2025-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0317387
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