Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
Titel:
Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
Auteur:
Ellison, Stuart M. Liao, Aiyin Wood, Shaun Taylor, Jessica Youshani, Amir Saam Rowlston, Sam Parker, Helen Armant, Myriam Biffi, Alessandra Chan, Lucas Farzaneh, Farzin Wynn, Rob Jones, Simon A. Heal, Paul Gaspar, H. Bobby Bigger, Brian W.