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An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model.
Peviani, Marco; Das, Sabyasachi; Patel, Janki; Jno-Charles, Odella; Kumar, Rajesh; Zguro, Ana; Mathews, Tyler D; Cabras, Paolo; Milazzo, Rita; Cavalca, Eleonora; Poletti, Valentina; Biffi, Alessandra.
Afiliación
  • Peviani M; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
  • Das S; Harvard Medical School, Boston, MA, USA.
  • Patel J; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy.
  • Jno-Charles O; Department of Biology and Biotechnology "L. Spallanzani", University of Pavia, Pavia, Italy.
  • Kumar R; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
  • Zguro A; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
  • Mathews TD; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
  • Cabras P; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
  • Milazzo R; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
  • Cavalca E; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
  • Poletti V; Department of Biology and Biotechnology "L. Spallanzani", University of Pavia, Pavia, Italy.
  • Biffi A; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy.
EMBO Mol Med ; 15(4): e15968, 2023 04 11.
Article en En | MEDLINE | ID: mdl-36876653
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Encéfalo / Sistema Nervioso Central Tipo de estudio: Prognostic_studies Límite: Animals Idioma: En Revista: EMBO Mol Med Asunto de la revista: BIOLOGIA MOLECULAR Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Encéfalo / Sistema Nervioso Central Tipo de estudio: Prognostic_studies Límite: Animals Idioma: En Revista: EMBO Mol Med Asunto de la revista: BIOLOGIA MOLECULAR Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos