论文信息 - CHAMPIONS: A phase 1/2 clinical trial with dose escalation of SB-913 ZFN-mediated in vivo human genome editing for treatment of MPS II (Hunter syndrome) - 字舞流文

CHAMPIONS: A phase 1/2 clinical trial with dose escalation of SB-913 ZFN-mediated in vivo human genome editing for treatment of MPS II (Hunter syndrome)

B. Burton | J. Muenzer | C. Whitley | P. Harmatz | C. Ficicioglu | C. W. P. Foo | C. Prada | H. Lau | S. Vaidya