AAV-Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: Enhancement of Immune Protection by Capsid Bioengineering

The goal of this project is to develop and test in vitro and in vivo hybrid recombinant adeno-associated viral vectors (rAAVs) covalently coated with tailored oligopeptide functionalized poly beta-amino esters (OM-PBAES) and/or zwitterionic polymers specifically designed to target muscular cells and to decrease the immune clearance, enabling a decrease in the effective dose and opening the possibility of redosage.

The project’s specific aims are (1) to generate hybrid polymer coated viruses, (2) to screen the polymer coated AAVs in vitro, and (3) to test the polymer coated AAVs in vivo and select the candidate showing the most effective immune protection and the highest muscle functional improvement.