Enhancing ZFN expression and nuclease activity in a genome editing construct leads to improvement of an in vivo genome editing platform

T. Wechsler,R. Dekelver, R. Radeke, L. Makani, Y. Santiago,I. Krivega,A. Conway, S. Sproul, H. Richards, Y. Bendana, D. Shivak, J. Miller,E. Rebar,M. C. Holmes

HUMAN GENE THERAPY(2018)

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