First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B
Titel:
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B
Auteur:
Harmatz, Paul Prada, Carlos E. Burton, Barbara K. Lau, Heather Kessler, Craig M. Cao, Liching Falaleeva, Marina Villegas, Andres G. Zeitler, Jennifer Meyer, Kathleen Miller, Weston Wong Po Foo, Cheryl Vaidya, Sagar Swenson, Wendy Shiue, Lisa H. Rouy, Didier Muenzer, Joseph