In vivo genome editing via non-viral delivery of zinc finger nucleases enables supraphysiological levels of therapeutic proteins and greater than 90% protein knockdown of multiple therapeutic gene targets via targeted integration and NHEJ, respectively, in wild type mice

A. Conway,M. Mendel, J. Garnes,A. Boyko,K. Kim,L. Zhang, R. C. DeKelver, D. E. Paschon, B. L. Mui,P. Lin,Y. K. Tam,C. Barbosa, T. Redelmeier,M. C. Holmes,G. Lee

HUMAN GENE THERAPY(2017)

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