ATFs 2.0: UBE3A activation without SNORD inhibition

Primary Investigator: D. Segal

Dr. Segal’s past work on Artificial transcription factors (ATFs) showed global paternal gene activation after subcutaneous injection. In bringing this therapeutic platform to the next level for human use it was prudent to evaluate options to find a mechanism of ensuring that we do not inhibit the SNORD116 and SNORD115 RNAs, which are associated with Prader -Willi syndrome. This proposal is to build on the capabilities of epigenetic editing and brain delivery seeking new targeting strategies to protect SNORD116.