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
Program History
In October 2023, the National Institutes of Health awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases.
The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome. The project team named under the grant includes representatives from Dr. Yong-Hui Jiang’s team and Dr. Jiangbing Zhou’s team at Yale, Dr. Elizabeth Berry-Kravis’ team at RUSH, and Dr. Allyson Berent and Jennifer Panagoulias from the Foundation for Angelman Syndrome Therapeutics (FAST).
The grant, from the NIH Common Fund, is part of a two-phase program with a total proposed budget of approximately $40 million over 5 years. The first phase, worth $26.5 million, is dedicated to generating all preclinical data and other key components towards a clinical trial for both diseases. The second phase, worth approximately $13.4 million, is intended to fund the Phase 1/2 clinical trial itself. The award of this second phase is contingent upon successful completion of the first phase.
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