About the Program
A gene editing tool that utilizes a guide RNA (gRNA) to recognize a specific section of DNA or RNA and directs an enzyme (nuclease) to cut at a specific section in the DNA or RNA. Designing a CRISPR that affects the UBE3A-ATS could potentially allow the paternal UBE3A gene to be turned on. It could also be used to edit a mutation in a gene, known as base editing.
- FAST Pillar
- Pillar 2: Turn on Dad’s UBE3A
- Company Website
- Zylka Lab