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.