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We develop therapeutics (gene therapies, ASO, small molecules, CRISPR, etc.) to treat single gene neurodevelopmental disorders such as Angelman syndrome, Pitt-Hopkins syndrome, and Dup15q syndrome. We also work to treat a vision disorder–IMPG2-mediated retinitis pigmentosa.


UNC AFFILIATIONS:

Carolina Institute for Developmental Disabilities (CIDD), Cell Biology & Physiology, Neuroscience Center

CLINICAL/RESEARCH INTERESTS:

Autism, Behavior, Bioinformatics, Biomedical Engineering, Cell Biology, Drug Delivery, Drug Discovery, Epigenetics and Chromatin Biology, Gene Therapy, Genetics, Microscopy, Molecular Biology, Molecular Medicine, Neurobiology, Pediatrics, Pharmacology, Translational Medicine