Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease
出生后早期进行 AAV9 基因治疗对 CLN3 疾病安全有效
期刊:Frontiers in Genetics
影响因子:2.8
doi:10.3389/fgene.2023.1118649
Tyler B Johnson, Jon J Brudvig, Shibi Likhite, Melissa A Pratt, Katherine A White, Jacob T Cain, Clarissa D Booth, Derek J Timm, Samantha S Davis, Brandon Meyerink, Ricardo Pineda, Cassandra Dennys-Rivers, Brian K Kaspar, Kathrin Meyer, Jill M Weimer
