Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector.
Seymour BJ, Singh S, Certo HM, Sommer K, Sather BD, Khim S, Clough C, Hale M, Pangallo J, Ryu BY, Khan IF, Adair JE, Rawlings DJ
Mol Ther Methods Clin Dev. 2021 Jan 20;20:635-651. doi: 10.1016/j.omtm.2021.01.007. eCollection 2021 Mar 12.
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