Funding Research for Treatments and a Cure

Finding new ways of Unsilencing Ube3a with CRISPR/Cas9
← Back to Search

Dr. Mark Zylka UNC-Chapel Hill
2016

Finding new ways of Unsilencing Ube3a with CRISPR/Cas9

$200,000

Based on previous research conducted at Dr. Mark Zylka’s lab and Dr. Ben Philpot’s lab, Dr. Zylka’s team will continue this work with the goal of unsilencing the paternal copy of Ube3a using new methods. Past research has shown that topoisomerase inhibitors, a drug compound, can unsilence the paternal copy of Ube3a, but that this drug also affects a number of other genes. Dr. Zylka’s team will use new technology (CRISPR/Cas9 screening technology) and a new approach to unsilence Ube3a, called a genetic screen. We know there are more than 20,000 genes in the human genome, and this genetic screen will evaluate each of those genes to see if any of them can unsilence the paternal copy of Ube3a. This is a novel new approach that has not been conducted before. In the end, the research team hopes to have a list of genes that can unsilence paternal Ube3a with little to no impact on other genes during the process.

Why this research is important:

We know from previous research that unsilencing Ube3a using specific drugs can affect a number of other genes in the process.
This research, using CRISPR/Cas9 screening technology and a new approach to unsilencing Ube3a, will look for genes—rather than drugs—that can unsilence paternal Ube3a with little to no impact on other genes during the process.

 
 

See more about CRISPR/CAS9 technology.