gene therapy – Angelman Syndrome Foundation

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 […]

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 […]

articles and announcements

Tag: gene therapy

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

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