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Induction and Tolerization of Dystrophin Immunity

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[Technical Report, Annual Report]

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Duchenne muscular dystrophy DMD, the most common form of muscular dystrophy, is a lethal X-linked recessive disorder caused by the dysfunction of a protein called dystrophin. Our growing understanding of DMD has led to an accurate characterization of this genetic disease yet an effective cure remains to be found. Therefore, there have been tremendous efforts from the scientific community to slow down or halt disease progression in DMD. Dystrophin gene therapy is an attractive avenue that has yet to overcome the challenge posed by dystrophin immunity an elicited immune response when restoring dystrophin. Herein, we obtained promising results that demonstrate that we can recapitulate this unwanted immune response in a dystrophic mouse model. A better characterization of this immune response is paramount to identify selective regulatory targets to dampen dystrophin immunity.


Subject Categories:

  • Medicine and Medical Research
  • Genetic Engineering and Molecular Biology

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[A, Approved For Public Release]