Induction and Tolerization of Dystrophin Immunity
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. Promising clinical gene therapies to restore dystrophin function have to overcome the immune barrier to a foreign protein. Herein, by generating a new mouse model, this proposal will target dystrophin immunity role in dystrophic corrective therapy. The expected outcomes are clear: (1) provide the community with a novel preclinical model to study this very specific immune barrier; (2) identify putative targets that could block this process that leads to dystrophin immunity; and (3) propose a safe and effective therapy alongside the dystrophin gene delivery to ensure successful tolerance to dystrophin and its restoration.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Nov 19, 2019
- Source ID
- W81XWH1910012
Entities
People
- Gérald Coulis
Organizations
- United States Army
- University of California, Irvine