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Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
An, Ding; Schneller, Jessica L; Frassetto, Andrea; Liang, Shi; Zhu, Xuling; Park, Ji-Sun; Theisen, Matt; Hong, Sue-Jean; Zhou, Jenny; Rajendran, Raj; Levy, Becca; Howell, Rebecca; Besin, Gilles; Presnyak, Vladimir; Sabnis, Staci; Murphy-Benenato, Kerry E; Kumarasinghe, E Sathyajith; Salerno, Timothy; Mihai, Cosmin; Lukacs, Christine M; Chandler, Randy J; Guey, Lin T; Venditti, Charles P; Martini, Paolo G V.
Afiliación
  • An D; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Schneller JL; Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD 20892, USA.
  • Frassetto A; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Liang S; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Zhu X; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Park JS; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Theisen M; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Hong SJ; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Zhou J; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Rajendran R; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Levy B; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Howell R; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Besin G; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Presnyak V; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Sabnis S; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Murphy-Benenato KE; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Kumarasinghe ES; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Salerno T; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Mihai C; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Lukacs CM; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Chandler RJ; Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD 20892, USA.
  • Guey LT; Moderna Therapeutics, Cambridge, MA 02139, USA.
  • Venditti CP; Organic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, NIH, Bethesda, MD 20892, USA. Electronic address: venditti@mail.nih.gov.
  • Martini PGV; Moderna Therapeutics, Cambridge, MA 02139, USA. Electronic address: paolo.martini@modernatx.com.
Cell Rep ; 21(12): 3548-3558, 2017 Dec 19.
Article en En | MEDLINE | ID: mdl-29262333
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: ARN Mensajero / Terapia Genética / Nanopartículas / Errores Innatos del Metabolismo de los Aminoácidos / Metilmalonil-CoA Mutasa Límite: Animals / Female / Humans / Male Idioma: En Revista: Cell Rep Año: 2017 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: ARN Mensajero / Terapia Genética / Nanopartículas / Errores Innatos del Metabolismo de los Aminoácidos / Metilmalonil-CoA Mutasa Límite: Animals / Female / Humans / Male Idioma: En Revista: Cell Rep Año: 2017 Tipo del documento: Article País de afiliación: Estados Unidos