CORDIS Project
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This project develops a novel CRISPR-based tool to enhance gene expression for treating genetic disorders related to neuronal excitability, particularly in epilepsy. It aims to address haploinsufficiency in specific genes to improve therapeutic outcomes.
This proposal will develop a new tool to rescue haploinsufficiency, which underlies many genetic disorders of neuronal excitability, and provide a new approach to treatment of intractable epilepsy.
This new tool is based on the CRISPR-On technology which can regulate the expression of endogenous genes by directly targeting their promoters, which allows expression of the full panoply of splice variants and untranslated regulatory sequences.
Importantly, the method does not require integrating exo…
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