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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.
Ou, Li; DeKelver, Russell C; Rohde, Michelle; Tom, Susan; Radeke, Robert; St Martin, Susan J; Santiago, Yolanda; Sproul, Scott; Przybilla, Michael J; Koniar, Brenda L; Podetz-Pedersen, Kelly M; Laoharawee, Kanut; Cooksley, Renee D; Meyer, Kathleen E; Holmes, Michael C; McIvor, R Scott; Wechsler, Thomas; Whitley, Chester B.
Afiliação
  • 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.
Mol Ther ; 27(1): 178-187, 2019 01 02.
Article em En | MEDLINE | ID: mdl-30528089

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Mucopolissacaridose I / Edição de Genes / Nucleases de Dedos de Zinco Limite: Animals Idioma: En Ano de publicação: 2019 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Mucopolissacaridose I / Edição de Genes / Nucleases de Dedos de Zinco Limite: Animals Idioma: En Ano de publicação: 2019 Tipo de documento: Article