CORDIS Project
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This project focuses on developing a new treatment for Wiedemann-Steiner Syndrome, a neurodevelopmental disorder, by using antisense oligonucleotides to up-regulate gene expression. The approach aims to create a fusion transcript that could alleviate disease symptoms, advancing both scientific understanding and potenti…
There is an immense interest in RNA therapeutics for a wide variety of health conditions.
Neurodevelopmental disorders (NDDs), which mostly have no available treatments or cures, affect millions of children and adults across Europe, and are estimated to account for as much as 1-3% of the total EU healthcare expenditure.
Wiedemann-Steiner Syndrome (WSS) is an NDD that currently has no available treatment and is not amenable to traditional gene therapy approaches.
Antisense oligonucleotides (ASOs)…
WEIZMANN INSTITUTE OF SCIENCE
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