articles and announcements

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

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 $2.8 million to continue the research.

Today, the findings of the initial studies have been published in Nature and described as “laying the groundwork for a long-lasting treatment or cure” not only for AS but potentially other single-gene disorders.

See the UNC press release for details