First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B
Paul Harmatz, Carlos E. Prada, Barbara K. Burton, Heather Lau, Craig M. Kessler, Liching Cao, Marina Falaleeva, Andres G. Villegas, Jennifer Zeitler, Kathleen Meyer, Weston P. Miller, Cheryl Wong Po Foo, Sagar A. Vaidya, Wendy Swenson, Lisa H. Shiue, Didier Rouy, Joseph Muenzer
Topics & Concepts
Genome editingZinc finger nucleaseZinc fingerIn vivoVirologyMucopolysaccharidosisBiologyGenomeGeneticsGeneBiochemistryTranscription factorCRISPR and Genetic EngineeringVirus-based gene therapy researchCAR-T cell therapy research