Current Appointments & Affiliations
Assistant Professor in Pediatrics
·
2024 - Present
Pediatrics, Pulmonary and Sleep Medicine,
Pediatrics
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 CiteGAA deficiency disrupts distal airway cells in Pompe disease.
Journal Article Am J Physiol Lung Cell Mol Physiol · September 1, 2023 Pompe disease is an autosomal recessive glycogen storage disease caused by mutations in the gene that encodes acid alpha-glucosidase (GAA)-an enzyme responsible for hydrolyzing lysosomal glycogen. GAA deficiency results in systemic lysosomal glycogen accum ... Full text Link to item CiteCross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing.
Journal Article Nat Commun · October 10, 2022 Recombinant adeno-associated viral (AAV) vectors are a promising gene delivery platform, but ongoing clinical trials continue to highlight a relatively narrow therapeutic window. Effective clinical translation is confounded, at least in part, by difference ... Full text Link to item CiteRecent Grants
Respiratory Characterization of DMD Mouse Models
ResearchPost Doctoral Trainee · Awarded by Sarepta Therapeutics, Inc. · 2019 - 2027Novel Adjunctive Therapies for Pompe Disease
ResearchPrincipal Investigator · Awarded by National Heart, Lung, and Blood Institute · 2024 - 2027Novel Adjunctive Therapies for Pompe Disease
ResearchPrincipal Investigator · Awarded by National Institutes of Health · 2022 - 2023View All Grants
Education, Training & Certifications
University of Florida, College of Medicine ·
2017
Ph.D.
University of Florida ·
2012
B.S.