In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.
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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.