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IMPROVING THE EFFECTIVENESS AND SAFETY OF EPIGENETIC EDITING IN BRAIN REGENERATION

Project description

Reprogramming brain cells to repair neurological damage

The brain has a very limited capacity to repair itself following injury or disease. Replacing lost neurons after stroke or neurodegeneration remains one of medicine’s greatest unmet needs. A promising strategy is to directly reprogramme glial cells – support cells already present in the brain – into functional neurons. The EIC-funded REGENERAR project is working to develop a non-viral delivery system to introduce epigenetic reprogramming agents into glial cells in the brain. Researchers will evaluate safety, targeting efficiency and reprogramming efficacy in cell cultures, brain tissue models and animals. Toxicological evaluation will be used to validate the readiness of the technology.

Objective

The central nervous system has a limited capacity for self-repair. Therefore, technologies to replace lost neurons after an injury, including stroke or neurodegenerative diseases, are of great need. The project REGENERAR aims to develop a non-viral delivery formulation up to TRL4 for the delivery of an epigenetic reprogramming formulation to reprogram glial cells into neurons. The in vitro safety and targeting of the formulation will be tested against glial cells and organotypic cortical slice cultures. Several routes of administrations will be tested to enhance the accumulation of the formulation in the brain. In addition, the in vivo safety, elimination, targeting and epigenetic reprogramming will be evaluated. In addition, toxicological studies will be performed in GLP conditions to evaluate both in vivo systemic and local (brain) effects of the formulation. The development of the non-viral formulation will take in account the contributes of key stakeholders in the area of gene editing, health institutions, patient associations, among others. A roadmap will be developed for upscaling innovation to higher TRL´s upon the end of the action.

Fields of science (EuroSciVoc)

Keywords

Programme(s)

Topic(s)

Funding Scheme

HORIZON-EIC - HORIZON EIC Grants

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Call for proposal

(opens in new window) HORIZON-EIC-2023-PATHFINDEROPEN-01

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Coordinator

UNIVERSIDADE DE COIMBRA
Net EU contribution
€ 1 100 000,00
Address
PACO DAS ESCOLAS
3004-531 Coimbra
Portugal
Activity type
Higher or Secondary Education Establishments
Links
Total cost
€ 1 100 000,00

Participants (5)