ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.

Autor: Ou L; Gene Therapy Center, University of Minnesota, Minneapolis, MN, USA., DeKelver RC; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., Rohde M; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., Tom S; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., Radeke R; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., St Martin SJ; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., Santiago Y; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., Sproul S; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., Przybilla MJ; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA., Koniar BL; Research Animal Resources, University of Minnesota, Minneapolis, MN, USA., Podetz-Pedersen KM; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA., Laoharawee K; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA., Cooksley RD; Gene Therapy Center, University of Minnesota, Minneapolis, MN, USA., Meyer KE; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., Holmes MC; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., McIvor RS; Center for Genome Engineering, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, USA., Wechsler T; Sangamo Therapeutics, Inc., 501 Canal Boulevard, Richmond, CA, USA., Whitley CB; Gene Therapy Center, University of Minnesota, Minneapolis, MN, USA. Electronic address: whitley@umn.edu.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2019 Jan 02; Vol. 27 (1), pp. 178-187. Date of Electronic Publication: 2018 Nov 01.