Update on phase 1/2 clinical trials for MPS I and MPS II using ZFN-mediated in vivo genome editing
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B. Burton | N. Leslie | J. Muenzer | C. Whitley | P. Harmatz | C. Ficicioglu | C. W. P. Foo | H. Lau | T. Wechsler | S. Vaidya | Edward Conner