In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model
使用新型 AAV-PHP 变体进行体内基因组编辑可挽救 SOD1-ALS 小鼠模型中的运动功能缺陷并延长生存期
期刊:Gene Therapy
影响因子:4.6
doi:10.1038/s41434-022-00375-w
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 Cullen, Galina Marsh, Stefan Hamann