Federal Project Grant Award Summary

The National Heart, Lung, and Blood Institute (NHLBI) awarded a $735,071 Project Grant to Indiana University Indianapolis on July 15, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837). The grant supports research and development of next-generation dystrophin gene therapy for Duchenne Muscular Dystrophy (DMD), with completion targeted for March 31, 2030. The project deliverables include development of an improved triple vector system utilizing protein trans-splicing technology and myotropic adeno-associated virus (AAV) capsid engineering to systemically deliver full-length dystrophin into striated muscle tissue. The research aims to overcome current clinical limitations of existing micro-dystrophin therapies by enabling safer, more effective systemic delivery with reduced AAV vector dosing and improved protection against dangerous host immune responses and organ toxicity.

The grant-funded research addresses critical efficacy and safety challenges identified in prior proof-of-concept studies, specifically targeting inefficient restoration of dystrophin in diaphragm muscles and potential immunogenicity concerns associated with dystrophin-intein fusion products. By advancing this gene therapy approach from animal models toward clinical translation, the project is expected to deliver improved therapeutic outcomes for DMD patients, over 90 percent of whom develop cardiomyopathy and face heart failure as a leading cause of mortality.