Enhancing Delivery of AAV-CRISPR to Muscle Stem Cells
Abstract
In this proposal, we will further develop our product, AAV9-CRISPR-DYS< delta-45-55>, a gene editing platform that permanently removes genetic mutations for approximately half of all Duchenne muscular dystrophy patients. Since Duchenne has no cure and only limited treatments, AAV9-CRISPR-DYS< delta-45-55> offers a potential therapy for a large cohort of Duchenne patients in about 5 years and offers permanent cure, as we are targeting the endogenous muscle stem cell called the satellite cell (SC). Additionally, the knowledge that will be obtained about SC targeting using AAV9-CRISPR-DYS< delta-45-55> will have far-reaching implications for improving the efficacy and safety of other AAV-based SC targeting therapies.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Dec 28, 2022
- Source ID
- W81XWH2210756
Entities
People
- April Pyle
Organizations
- United States Army
- University of California, Los Angeles