Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease
慢病毒造血干细胞基因治疗挽救庞贝病小鼠模型的临床表型
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2020.07.001
Giuseppa Piras, Claudia Montiel-Equihua, Yee-Ka Agnes Chan, Slawomir Wantuch, Daniel Stuckey, Derek Burke, Helen Prunty, Rahul Phadke, Darren Chambers, Armando Partida-Gaytan, Diego Leon-Rico, Neelam Panchal, Kathryn Whitmore, Miguel Calero, Sara Benedetti, Giorgia Santilli, Adrian J Thrasher, H Bob
