Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa)
DTX401(一种AAV8介导的肝脏靶向基因疗法)在I型糖原贮积症(GSDIa)成人患者中的安全性和有效性
期刊:Journal of Inherited Metabolic Disease
影响因子:3.8
doi:10.1002/jimd.70014
Weinstein, David A; Derks, Terry G; Rodriguez-Buritica, David F; Ahmad, Ayesha; Couce, María-Luz; Mitchell, John J; Riba-Wolman, Rebecca; Mount, Malaya; Sallago, Julieta Bonvin; Ross, Katalin M; van der Klauw, Melanie M; de Boer, Foekje; van der Schaaf, Caroline; Saavedra, Heather; Martínez-Olmos, Miguel; Atanga, Elvis; Hosseini, Asad; Mitragotri, Deepali; Crombez, Eric