Ben Philpot, PhD and his lab at UNC Chapel Hill have identified a small molecule that could be a potential gene therapy treatment for AS. The ASF first funded Dr. Philpot’s pilot study to partner with Pfizer to evaluate over […]
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As part of a 2020 ASF funded study, Ben Philpot, PhD and Matt Judson, PhD at UNC School of Medicine have been developing a gene therapy strategy as a method to restore function of UBE3A. The early results were published […]
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Mark Zylka, PhD and his team at UNC School of Medicine have been studying CRISPR-Cas9 as a method to unsilence the paternal copy of UBE3A. After ASF funded the project, the findings were so positive, the NIH provided additional funding of […]
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