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ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice.
Pagant, Silvere; Huston, Marshall W; Moreira, Luciana; Gan, Lin; St Martin, Susan; Sproul, Scott; Holmes, Michael C; Meyer, Kathleen; Wechsler, Thomas; Desnick, Robert J; Yasuda, Makiko.
Afiliación
  • Pagant S; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
  • Huston MW; Sangamo Therapeutics, Inc., Brisbane, CA 94005, USA.
  • Moreira L; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
  • Gan L; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
  • St Martin S; Sangamo Therapeutics, Inc., Brisbane, CA 94005, USA.
  • Sproul S; Sangamo Therapeutics, Inc., Brisbane, CA 94005, USA.
  • Holmes MC; Sangamo Therapeutics, Inc., Brisbane, CA 94005, USA.
  • Meyer K; Sangamo Therapeutics, Inc., Brisbane, CA 94005, USA.
  • Wechsler T; Sangamo Therapeutics, Inc., Brisbane, CA 94005, USA.
  • Desnick RJ; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
  • Yasuda M; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA. Electronic address: makiko.yasuda@mssm.edu.
Mol Ther ; 29(11): 3230-3242, 2021 11 03.
Article en En | MEDLINE | ID: mdl-33775910
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad de Fabry / Alfa-Galactosidasa / Hepatocitos / Edición Génica / Nucleasas con Dedos de Zinc Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad de Fabry / Alfa-Galactosidasa / Hepatocitos / Edición Génica / Nucleasas con Dedos de Zinc Límite: Animals / Humans Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos