Targeting the dark side of the human genome—non-coding regulatory elements—to treat diseases

Introduction

The 98% of the human genome, sometimes referred to as the dark side of the human genome, contains many non-coding regulatory elements (NCREs), which control the combinational expression of the limited number of genes (only 2% of the genome) to form diverse cell types and tissues of a complex human body, all from the identical copy of the genome.

Genetic Variants and Therapeutic Targets

Most genetic variants and mutations also lie in NCREs. However, the number of NCREs as potential therapeutic targets remains very limited.

Project Overview

In TargetNCREs, two novel and unique high-throughput screening systems further developed within the ECR starting grant (Silencer) will be exploited for their innovation and commercial potential to harness the therapeutic values of NCREs.

Objectives

We aim to:

1. Test and validate previously identified top hits in vivo.
2. Carry out pilot research to show the technologies to be effective and appropriate for commercial applications.
3. Clarify the IPR protection strategy.
4. Engage partners and stakeholders to focus on potential pipelines.
5. Perform a market search for future business development.