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Opti-AAV

Financiado

Towards a gene therapy for age-related macular degeneration (AMD)

While the blood neural barriers represent significant hurdles for the development of gene therapies for neurological and ophthalmological conditions it is now becoming clear that many of these conditions actually present with disr... ver más

31/12/2023

TRINITY COLLEGE DU...

150K€

Presupuesto del proyecto: 150K€

Líder del proyecto

THE PROVOST FELLOWS FOUNDATION SCHOLARS THE... No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Financiación concedida El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2023-12-31

ERC-2022-POC1: ERC PROOF OF CONCEPT GRANTS1 Objective:Objectives:

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Cerrada hace 3 años

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1 Participantes

TRINITY COLLEGE DUBLIN

150.00K€ | Lider

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Duración del proyecto: 19 meses Fecha Inicio: 2022-05-31
Fecha Fin: 2023-12-31

Líder del proyecto

THE PROVOST FELLOWS FOUNDATION SCHOLARS THE... No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 150K€

Descripción del proyecto While the blood neural barriers represent significant hurdles for the development of gene therapies for neurological and ophthalmological conditions it is now becoming clear that many of these conditions actually present with disrupted barrier integrity as the driving force of the pathology. Included in these diseases is age related macular degeneration (AMD), in which we have shown that the blood retina barrier (BRB) is sub-clinically disrupted and is a key driving force for disease progression (ERC funded Retina Rhythm project). Specifically related to this Proof of Concept grant, a novel form of therapy for the end stage of AMD, namely geographic atrophy (GA), is now desperately needed. A targeted approach to locally delivering adeno associated virus (AAV) to endothelial cells of the BRB would offer the opportunity to restore barrier function and prevent disease. However, the current tools available to achieve this localized approach to gene therapy are not optimum. Here we will use an exosome encapsulated and endothelial cell specific AAV to achieve robust transduction of retinal endothelial cells. This modified and optimized AAV (Opti-AAV) will represent a novel tool in translating recently identified biological mechanisms into real and meaningful therapies for patients.

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