Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy Arsa Knock-Out Mice
单次系统性基因疗法治疗异染性脑白质营养不良症 Arsa 基因敲除小鼠的疾病
期刊:Journal of Neuroscience
影响因子:4.4
doi:10.1523/JNEUROSCI.1829-22.2023
Thia St Martin, Tania A Seabrook, Katherine Gall, Jenn Newman, Nancy Avila, April Hayes, Monicah Kivaa, Jason Lotterhand, Michael Mercaldi, Kruti Patel, Israel J Rivas, Stephen Woodcock, Teresa L Wright, Albert B Seymour, Omar L Francone, Jacinthe Gingras