Immune-mediated pathology in Duchenne muscular dystrophy

Amy S. Rosenberg; Montserrat Puig; Kanneboyina Nagaraju; Eric P. Hoffman; S. Armando Villalta; V. Ashutosh Rao; Lalage M. Wakefield; Janet Woodcock

doi:10.1126/scitranslmed.aaa7322

Immune-mediated pathology in Duchenne muscular dystrophy

Amy S. Rosenberg
, Montserrat Puig
, Kanneboyina Nagaraju
, Eric P. Hoffman
, S. Armando Villalta
, V. Ashutosh Rao
, Lalage M. Wakefield
, Janet Woodcock

Binghamton University

United States Food and Drug Administration
University of California at Irvine
National Institutes of Health

Research output: Contribution to journal › Review article › peer-review

253 Scopus citations

Abstract

Immunological and inflammatory processes downstream of dystrophin deficiency as well asmetabolic 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.

Original language	English
Article number	299rv4
Journal	Science Translational Medicine
Volume	7
Issue number	299
DOIs	https://doi.org/10.1126/scitranslmed.aaa7322
State	Published - Aug 5 2015

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title = "Immune-mediated pathology in Duchenne muscular dystrophy",

abstract = "Immunological and inflammatory processes downstream of dystrophin deficiency as well asmetabolic 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.",

author = "Rosenberg, \{Amy S.\} and Montserrat Puig and Kanneboyina Nagaraju and Hoffman, \{Eric P.\} and Villalta, \{S. Armando\} and Rao, \{V. Ashutosh\} and Wakefield, \{Lalage M.\} and Janet Woodcock",

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doi = "10.1126/scitranslmed.aaa7322",

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T1 - Immune-mediated pathology in Duchenne muscular dystrophy

AU - Rosenberg, Amy S.

AU - Puig, Montserrat

AU - Nagaraju, Kanneboyina

AU - Hoffman, Eric P.

AU - Villalta, S. Armando

AU - Rao, V. Ashutosh

AU - Wakefield, Lalage M.

AU - Woodcock, Janet

PY - 2015/8/5

Y1 - 2015/8/5

N2 - Immunological and inflammatory processes downstream of dystrophin deficiency as well asmetabolic 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.

AB - Immunological and inflammatory processes downstream of dystrophin deficiency as well asmetabolic 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.

UR - https://www.scopus.com/pages/publications/84938717678

U2 - 10.1126/scitranslmed.aaa7322

DO - 10.1126/scitranslmed.aaa7322

M3 - Review article

C2 - 26246170

SN - 1946-6234

VL - 7

JO - Science Translational Medicine

JF - Science Translational Medicine

IS - 299

M1 - 299rv4

ER -

Immune-mediated pathology in Duchenne muscular dystrophy

Abstract

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