Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
慢病毒载体介导的体外干细胞基因治疗治疗粘多糖贮积症IIIA的临床前安全性和有效性
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2019.04.001
Stuart M Ellison, Aiyin Liao, Shaun Wood, Jessica Taylor, Amir Saam Youshani, Sam Rowlston, Helen Parker, Myriam Armant, Alessandra Biffi, Lucas Chan, Farzin Farzaneh, Rob Wynn, Simon A Jones, Paul Heal, H Bobby Gaspar, Brian W Bigger