About the Program
Nucleoside analogs are synthetic molecules with a chemically altered backbone that helps them to resist breakdown by enzymes that normally degrade RNA-like molecules. They could be used to target the UBE3A-ATS, unsilencing the paternal copy of the UBE3A gene and enabling UBE3A protein to be made.
- FAST Pillar
- Pillar 2: Turn on Dad’s UBE3A
- Collaborators
Program History
Frances Arnold, PhD, winner of the 2018 Nobel Prize in Chemistry, has received FAST funding to develop new molecular tools that could help develop highly durable RNA-targeted therapies for Angelman syndrome.
The project will apply directed evolution, the Nobel Prize-winning approach pioneered by Dr. Arnold, to engineer enzymes capable of producing new nucleoside analogs with properties that may be useful to target the UBE3A antisense (UBE3A-ATS) transcript.
The grant is supported by Maddie’s Mission Foundation in partnership with the Shaw Family, an organization dedicated to transforming care for individuals living with Angelman syndrome and other rare neurological conditions.
Timeline:
Grant Awarded