About the Program
A therapeutic approach using small pieces of RNA, called divalent small interfering-RNA (di-siRNA) that are aimed to bind to the UBE3A-ATS and prevent the silencing of the paternal copy of the UBE3A gene, enabling UBE3A protein to be made.
- FAST Pillar
- Pillar 2: Turn on Dad’s UBE3A
- Collaborators
Mark Deehan