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Immune-mediated pathology in Duchenne muscular dystrophy.
Rosenberg, Amy S; Puig, Montserrat; Nagaraju, Kanneboyina; Hoffman, Eric P; Villalta, S Armando; Rao, V Ashutosh; Wakefield, Lalage M; Woodcock, Janet.
Afiliação
  • Rosenberg AS; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Building 71/2238, Silver Spring, MD 20993, USA. amy.rosenberg@fda.hhs.gov.
  • Puig M; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Building 71/2238, Silver Spring, MD 20993, USA.
  • Nagaraju K; Center for Genetic Medicine Research, Children's National Medical Center, Washington, DC 20010, USA.
  • Hoffman EP; Center for Genetic Medicine Research, Children's National Medical Center, Washington, DC 20010, USA.
  • Villalta SA; Department of Physiology and Biophysics, Institute for Immunology, University of California, Irvine, Irvine, CA 92697, USA.
  • Rao VA; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Building 71/2238, Silver Spring, MD 20993, USA.
  • Wakefield LM; Laboratory of Cancer Biology and Genetics, National Cancer Institute, Building 37, Room 4032A, Bethesda, MD 20892, USA.
  • Woodcock J; Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Building 71/2238, Silver Spring, MD 20993, USA.
Sci Transl Med ; 7(299): 299rv4, 2015 Aug 05.
Article em En | MEDLINE | ID: mdl-26246170
ABSTRACT
Immunological and inflammatory processes downstream of dystrophin deficiency as well as metabolic abnormalities, defective autophagy, and loss of regenerative capacity all contribute to muscle pathology in Duchenne muscular dystrophy (DMD). These downstream cascades offer potential avenues for pharmacological intervention. Modulating the inflammatory response and inducing immunological tolerance to de novo dystrophin expression will be critical to the success of dystrophin-replacement therapies. This Review focuses on the role of the inflammatory response in DMD pathogenesis and opportunities for clinical intervention.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne / Imunidade Inata Idioma: En Ano de publicação: 2015 Tipo de documento: Article
Texto completo
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XML
PubMed Links
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne / Imunidade Inata Idioma: En Ano de publicação: 2015 Tipo de documento: Article