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

Project description

Exploring the ‘dark side’ of human genome

There is a ‘dark side’ of the human genome that remains unknown. However, the 98 % of the genome including DNA sequences known as non-coding regulatory elements (NCREs) holds immense potential for understanding and treating genetic disorders yet remains largely untapped. With this in mind, the ERC-funded TargetNCREs project will build on the groundwork laid by the Silencer ECR starting grant. This initiative pioneers two innovative high-throughput screening systems. By identifying and validating therapeutic targets within NCREs, TargetNCREs aims to revolutionise precision medicine, paving the way for novel treatments and therapies. With a focus on commercialization and collaboration, this project holds promise for the future of genetic research and healthcare.

Objective

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. Most genetic variants and mutations also lie in NCREs. However, the number of NCREs as potential therapeutic targets remains very limited. 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. We aim to test and validate previously identified top hits in vivo, carry out pilot research to show the technologies to be effective and appropriate for commercial applications, clarify the IPR protection strategy, engage partners and stakeholders to focus on potential pipelines, and perform a market search for future business development.

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Topic(s)

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Funding Scheme

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HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants

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Call for proposal

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(opens in new window) ERC-2023-POC

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Host institution

ACADEMISCH ZIEKENHUIS LEIDEN
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 150 000,00
Address
ALBINUSDREEF 2
2333 ZA Leiden
Netherlands

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Activity type
Higher or Secondary Education Establishments
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Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

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Beneficiaries (1)

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