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Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment.
Ramos, Daniel M; d'Ydewalle, Constantin; Gabbeta, Vijayalakshmi; Dakka, Amal; Klein, Stephanie K; Norris, Daniel A; Matson, John; Taylor, Shannon J; Zaworski, Phillip G; Prior, Thomas W; Snyder, Pamela J; Valdivia, David; Hatem, Christine L; Waters, Ian; Gupte, Nikhil; Swoboda, Kathryn J; Rigo, Frank; Bennett, C Frank; Naryshkin, Nikolai; Paushkin, Sergey; Crawford, Thomas O; Sumner, Charlotte J.
Afiliación
  • Ramos DM; Department of Neuroscience and.
  • d'Ydewalle C; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Gabbeta V; PTC Therapeutics, South Plainfield, New Jersey, USA.
  • Dakka A; PTC Therapeutics, South Plainfield, New Jersey, USA.
  • Klein SK; Ionis Pharmaceuticals, Carlsbad, California, USA.
  • Norris DA; Ionis Pharmaceuticals, Carlsbad, California, USA.
  • Matson J; Ionis Pharmaceuticals, Carlsbad, California, USA.
  • Taylor SJ; PharmOptima, Portage, Michigan, USA.
  • Zaworski PG; PharmOptima, Portage, Michigan, USA.
  • Prior TW; Center for Human Genetics, Case Western Reserve University, Cleveland, Ohio, USA.
  • Snyder PJ; Department of Pathology, Ohio State University, Columbus, Ohio, USA.
  • Valdivia D; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Hatem CL; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Waters I; The Sidney Kimmel Comprehensive Cancer Center, Department of Oncology, and.
  • Gupte N; Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Swoboda KJ; Center for Genomic Medicine, Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  • Rigo F; Ionis Pharmaceuticals, Carlsbad, California, USA.
  • Bennett CF; Ionis Pharmaceuticals, Carlsbad, California, USA.
  • Naryshkin N; PTC Therapeutics, South Plainfield, New Jersey, USA.
  • Paushkin S; SMA Foundation, New York, New York, USA.
  • Crawford TO; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Sumner CJ; Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
J Clin Invest ; 129(11): 4817-4831, 2019 11 01.
Article en En | MEDLINE | ID: mdl-31589162
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Médula Espinal / Envejecimiento / Atrofia Muscular Espinal / Oligodesoxirribonucleótidos Antisentido / Neuronas Motoras Límite: Female / Humans / Male Idioma: En Revista: J Clin Invest Año: 2019 Tipo del documento: Article Pais de publicación: Estados Unidos
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Médula Espinal / Envejecimiento / Atrofia Muscular Espinal / Oligodesoxirribonucleótidos Antisentido / Neuronas Motoras Límite: Female / Humans / Male Idioma: En Revista: J Clin Invest Año: 2019 Tipo del documento: Article Pais de publicación: Estados Unidos