Accession Number:

AD1146185

Title:

Induction and Tolerization of Dystrophin Immunity

Descriptive Note:

[Technical Report, Annual Report]

Corporate Author:

CALIFORNIA UNIV IRVINE

Personal Author(s):

Report Date:

2021-07-01

Pagination or Media Count:

14

Abstract:

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.

Descriptors:

Subject Categories:

  • Medicine and Medical Research
  • Genetic Engineering and Molecular Biology

Distribution Statement:

[A, Approved For Public Release]