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PERSONAL WEBSITE:
https://www.med.unc.edu/cellbiophysio/directory/ben-philpot-phd/ |
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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.