Project Details
Description
The project is designed to generate preliminary data to support the feasibility of rescuing full-length SYNGAP1 protein in human induced pluripotent stem cell (hiPSC) derived cortical excitatory neurons that are heterozygous for a premature termination codon (nonsense) mutation. The approached used to rescue SYNGAP1 nonsense mutation involves use of anticodon edited transfer RNA (ACE-tRNA) technology.
| Status | Active |
|---|---|
| Effective start/end date | 9/1/21 → 6/30/23 |
Funding
- Soley for Syngap1 e.V. (AGMT - 10/29/2021)
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