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MCOLN1 gene therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV.
DeRosa, Samantha; Salani, Monica; Smith, Sierra; Sangster, Madison; Miller-Browne, Victoria; Wassmer, Sarah; Xiao, Ru; Vandenberghe, Luk; Slaugenhaupt, Susan; Misko, Albert; Grishchuk, Yulia.
  • DeRosa S; Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute/Harvard Medical School, Boston, MA 02114, USA.
  • Salani M; Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute/Harvard Medical School, Boston, MA 02114, USA.
  • Smith S; Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute/Harvard Medical School, Boston, MA 02114, USA.
  • Sangster M; Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute/Harvard Medical School, Boston, MA 02114, USA.
  • Miller-Browne V; Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute/Harvard Medical School, Boston, MA 02114, USA.
  • Wassmer S; Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary and Harvard Medical School, Boston, MA 02114, USA.
  • Xiao R; Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary and Harvard Medical School, Boston, MA 02114, USA.
  • Vandenberghe L; Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary and Harvard Medical School, Boston, MA 02114, USA.
  • Slaugenhaupt S; Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute/Harvard Medical School, Boston, MA 02114, USA.
  • Misko A; Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute/Harvard Medical School, Boston, MA 02114, USA.
  • Grishchuk Y; Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute/Harvard Medical School, Boston, MA 02114, USA.
Hum Mol Genet ; 30(10): 908-922, 2021 05 29.
Article en En | MEDLINE | ID: mdl-33822942
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia Genética / Canales de Potencial de Receptor Transitorio / Mucolipidosis / Enfermedades del Sistema Nervioso Límite: Animals / Humans Idioma: En Año: 2021 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia Genética / Canales de Potencial de Receptor Transitorio / Mucolipidosis / Enfermedades del Sistema Nervioso Límite: Animals / Humans Idioma: En Año: 2021 Tipo del documento: Article