First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B
- Resource Type
- Article
- Authors
- 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
- Source
- In Molecular Therapy 7 December 2022 30(12):3587-3600
- Subject
- Language
- ISSN
- 1525-0016