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articles and announcements

Tag: gene therapy

July 8, 2024

Researchers at UNC Chapel Hill Find New Potential Treatment for AS

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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Ben Philpot, PhD
October 22, 2021

ASF Funded Research on Gene Therapy in AS Published in JCI Insight

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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Dr. Mark Zylka
October 21, 2020

Groundbreaking ASF Funded Research on Gene Therapy in AS Published in Nature

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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Recent Posts

  • First Patient(s) Dosed in Ionis Phase 3 ION582 Study
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  • First Patient(s) Dosed in Ultragenyx Phase 3 Aspire Study

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The mission of the Angelman Syndrome Foundation is to advance the awareness and treatment of Angelman syndrome through education and information, research, and support for individuals with Angelman syndrome, their families and other concerned parties. We exist to give all of them a reason to smile, with the ultimate goal of finding a cure.

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