Hugh Lee Sweeney

This paper develops and tests dual AAV vectors for treating USH1B by reconstituting full-length MYO7A expression, showing safety and efficacy in non-human primates.

This study generates a rat model of Duchenne muscular dystrophy using CRISPR/Cas9 and evaluates its functional and structural muscle pathologies across different ages.

This paper investigates dual S100A1 and ARC gene therapy for DMD cardiomyopathy, showing improved cardiac function and survival in mouse models and safety in canine models.

This paper develops a selective non-muscle myosin II inhibitor, MT-228, with high brain penetration and efficacy in preclinical models, offering therapeutic potential for diseases like stimulant use disorder.