First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
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B. Burton | C. Kessler | D. Rouy | J. Muenzer | M. Falaleeva | P. Harmatz | C. W. P. Foo | C. Prada | W. Miller | H. Lau | Kathleen E. Meyer | Lisa H. Shiue | S. Vaidya | L. Cao | A. G. Villegas | W. Swenson | Jennifer Zeitler