Project description
Gene therapy for age-related macular degeneration
Gene therapy for the eye involves the delivery of genetic material directly into retinal cells to treat or prevent eye diseases caused by genetic mutations. Gene therapies usually employ viral vectors to provide long-term benefits, reduce the need for frequent treatments and improve quality of life for patients with previously untreatable eye conditions. The ERC-funded Retina-Seal project focuses on age-related macular degeneration (AMD), the leading cause of irreversible central blindness. The project aims to enhance claudin-5 expression, a protein known to regulate the movement of molecules in and out of the retina and to cause vascular leakage. Researchers will develop novel adeno-associated viruses carrying the CLDN-5 gene, aiming to stabilise the blood-retina barrier and prevent AMD progression.
Objective
"Age-related macular degeneration (AMD) is the leading cause of irreversible central blindness in the world. The number of people with AMD is predicted to be 288 million by 2040, with an estimated 1 in 10 people over the age of 55 already showing early signs of the condition.
At the inner blood retina barrier (iBRB), claudin-5 is the most enriched component of the tight junctions of endothelial cells, regulating the paracellular diffusion of molecules into and out of the retina. As part of the on-going ERC Consolidator grant Retina-Rhythm, we now have a very large body of data suggesting “leaky” or “CLDN-5 depleted” retinal vasculature drives the accumulation of waste material known as drusen, the hallmark diagnostic feature of AMD. Therefore, methods aimed at enhancing CLDN-5 expression can potentially stabilize vascular integrity and prevent the onset of AMD (both dry and wet AMD). Here, we will develop novel adeno-associated viruses (AAV) that will directly regulate the expression of 2 novel target genes and effectively ""seal"" the blood retina barrier and stabilise disease progression."
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- natural sciences biological sciences microbiology virology
- medical and health sciences clinical medicine ophthalmology
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Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.1.1 - European Research Council (ERC)
MAIN PROGRAMME
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Topic(s)
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Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) ERC-2024-POC
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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.
D02 CX56 Dublin
Ireland
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.