Project Details
Description
CHD2 NMD exon targeting in patient and CRISPR generated cell lines. The initial proof of principle experiments is to establish whether targeting an NMD-inducing exon can restore CHD2 protein expression in haploinsufficient lines to levels similar to those in control individuals.
| Status | Finished |
|---|---|
| Effective start/end date | 9/5/19 → 9/5/20 |
Funding
- Stoke Therapeutics, Inc. (Agmt 9/5/19)
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