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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. Among authors: walleshauser j. bioRxiv [Preprint]. 2024 Dec 30:2024.12.30.630799. doi: 10.1101/2024.12.30.630799. bioRxiv. 2024. PMID: 39803585 Free PMC article. Preprint.
Identification of a Guide RNA Targeting an Ultraconserved Element for Evaluation of Cas9 Genome Editors Across Mammalian Species.
Gowen BG, Khekare P, McCawley SR, Melton K, Soares C, Chan J, Jani V, Boivin P, Bans A, Leong WI, Cantor AJ, Walleshauser J, Otoupal PB, Mepani RJ, Silverman AP, Haak-Frendscho M, Wei SC. Gowen BG, et al. Among authors: walleshauser j. CRISPR J. 2024 Dec;7(6):306-309. doi: 10.1089/crispr.2024.0053. Epub 2024 Sep 23. CRISPR J. 2024. PMID: 39308435 No abstract available.