Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease
- Resource Type
- Journal
- Authors
- Piras, Giuseppa; Montiel-Equihua, Claudia; Chan, Yee-Ka Agnes; Wantuch, Slawomir; Stuckey, Daniel; Burke, Derek; Prunty, Helen; Phadke, Rahul; Chambers, Darren; Partida-Gaytan, Armando; Leon-Rico, Diego; Panchal, Neelam; Whitmore, Kathryn; Calero, Miguel; Benedetti, Sara; Santilli, Giorgia; Thrasher, Adrian J.; Gaspar, H. Bobby
- Source
- MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT; SEP 11 2020, 18 p558-p570, 13p.
- Subject
- Language
- English
- ISSN
- 23290501