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A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases.
Ou, Li; Przybilla, Michael J; Tabaran, Alexandru-Flaviu; Overn, Paula; O'Sullivan, M Gerard; Jiang, Xuntian; Sidhu, Rohini; Kell, Pamela J; Ory, Daniel S; Whitley, Chester B.
  • Ou L; Gene Therapy Center, Department of Pediatrics, University of Minnesota, Minneapolis, MN, 55455, USA. ouxxx045@umn.edu.
  • Przybilla MJ; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN, 55455, USA.
  • Tabaran AF; Comparative Pathology Shared Resource, University of Minnesota Masonic Cancer Center, Saint Paul, MN, 55108, USA.
  • Overn P; Comparative Pathology Shared Resource, University of Minnesota Masonic Cancer Center, Saint Paul, MN, 55108, USA.
  • O'Sullivan MG; Comparative Pathology Shared Resource, University of Minnesota Masonic Cancer Center, Saint Paul, MN, 55108, USA.
  • Jiang X; Department of Medicine, Washington University School of Medicine, Saint Louis, MO, USA.
  • Sidhu R; Department of Medicine, Washington University School of Medicine, Saint Louis, MO, USA.
  • Kell PJ; Department of Medicine, Washington University School of Medicine, Saint Louis, MO, USA.
  • Ory DS; Department of Medicine, Washington University School of Medicine, Saint Louis, MO, USA.
  • Whitley CB; Gene Therapy Center, Department of Pediatrics, University of Minnesota, Minneapolis, MN, 55455, USA.
Gene Ther ; 27(5): 226-236, 2020 05.
Article en En | MEDLINE | ID: mdl-31896760

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedad de Sandhoff / Enfermedad de Tay-Sachs Tipo de estudio: Guideline Límite: Animals / Humans Idioma: En Año: 2020 Tipo del documento: Article

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedad de Sandhoff / Enfermedad de Tay-Sachs Tipo de estudio: Guideline Límite: Animals / Humans Idioma: En Año: 2020 Tipo del documento: Article