CHD2 NMD Exon Targeting in Patient and CRISPR-generated Cell Lines.

    Project: Research project

    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.
    StatusFinished
    Effective start/end date9/5/199/5/20

    Funding

    • Stoke Therapeutics, Inc. (Agmt 9/5/19)

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