Overview
Synthetic Virology & Gene Therapy
Current Appointments & Affiliations
Professor in Surgery
·
2019 - Present
Surgery, Surgical Sciences,
Surgery
Director of Gene Therapy
·
2018 - Present
Surgery, Surgical Sciences,
Surgery
Professor of Molecular Genetics and Microbiology
·
2019 - Present
Molecular Genetics and Microbiology,
Basic Science Departments
Affiliate of the Duke Regeneration Center
·
2021 - Present
Duke Regeneration Center,
Basic Science Departments
Recent Publications
GAA replacement improves respiratory muscle, neural, and alveolar pathology in the pompe mouse.
Journal Article Respir Physiol Neurobiol · July 2025 Pompe disease is a devastating neuromuscular disorder caused by mutations in the gene GAA. These mutations result in a deficiency of the enzyme acid α-glucosidase (GAA), leading to lysosomal glycogen accumulation in cardiac, skeletal, and smooth muscle, mo ... Full text Link to item CiteEvaluation of AAV Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene Therapy.
Journal Article Transl Stroke Res · June 2025 Nosebleeds and intracranial hemorrhage from brain arteriovenous malformations (bAVMs) are among the most devastating symptoms of patients with hereditary hemorrhagic telangiectasis (HHT). All available managements have limitations. We showed that intraveno ... Full text Link to item CiteDeletion of Exon 51 in a Humanized Duchenne Muscular Dystrophy Mouse Model Abolishes Ventricular Arrhythmia Predisposition.
Journal Article Circ Genom Precis Med · June 2025 Full text Link to item CiteRecent Grants
Enhancing limb regeneration with gene therapy informed by comparative biology
ResearchCollaborator · Awarded by National Institutes of Health · 2024 - 2029Optimizing Gene Therapy for Respiratory Insufficiency in Duchenne Muscular Dystrophy
ResearchCo Investigator · Awarded by National Institutes of Health · 2024 - 2028Determinants of AAV Tropism
ResearchPrincipal Investigator · Awarded by National Heart, Lung, and Blood Institute · 2008 - 2028View All Grants
Education, Training & Certifications
University of North Carolina, Chapel Hill ·
2004
Ph.D.