Immune-mediated pathology in Duchenne muscular dystrophy

AS Rosenberg, M Puig, K Nagaraju… - Science translational …, 2015 - science.org
AS Rosenberg, M Puig, K Nagaraju, EP Hoffman, SA Villalta, VA Rao, LM Wakefield…
Science translational medicine, 2015science.org
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 …
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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