In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.
Chen YA, Kankel MW, Hana S, Lau SK, Zavodszky MI, McKissick O, Mastrangelo N, Dion J, Wang B, Ferretti D, Koske D, Lehman S, Koszka K, McLaughlin H, Liu M, Marshall E, Fabian AJ, Cullen P, Marsh G, Hamann S, Craft M, Sebalusky J, Arnold HM, Driscoll R, Sheehy A, Luo Y, Manca S, Carlile T, Sun C, Sigrist K, McCampbell A, Henderson CE, Lo SC.
Chen YA, et al. Among authors: dion j.
Gene Ther. 2023 May;30(5):443-454. doi: 10.1038/s41434-022-00375-w. Epub 2022 Dec 1.
Gene Ther. 2023.
PMID: 36450833
Free PMC article.
We observed substantial protection against disease progression, demonstrating the utility of our CRISPR editing preclinical approach for target evaluation. Our approach uncovered key parameters (e.g., AAV capsid, Cas9 expression) that resulted in improved efficacy compared …
We observed substantial protection against disease progression, demonstrating the utility of our CRISPR editing preclinical approach for tar …