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

Gene Therapy, Published online: 01 December 2022; doi:10.1038/s41434-022-00375-wIn vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model

Source: Gene Therapy - December 1, 2022 Category: Genetics & Stem Cells Authors: Yi A. Chen Mark W. Kankel Sam Hana Shukkwan Kelly Lau Maria I. Zavodszky Olivia McKissick Nicole Mastrangelo Jessica Dion Bin Wang Daniel Ferretti David Koske Sydney Lehman Kathryn Koszka Helen McLaughlin Mei Liu Eric Marshall Attila J. Fabian Patrick Cul Source Type: research