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: zavodszky mi.
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.