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

Project description

Addressing barrier integrity issues in the treatment of age-related macular degeneration

The macula is the part of the eye that is responsible for central vision. Damage to the macula can gradually occur as an individual ages, resulting in vision problems that significantly impact daily life. Research into age-related macular degeneration (AMD) has shown that disrupted endothelial barrier integrity is a significant driving force behind this condition. Based on this discovery, scientists of the EU-funded Opti-AAV project are working towards a novel therapy approach for AMD that involves the local delivery of exosome-encapsulated endothelial cell-specific adeno-associated virus into the blood–retina barrier. The aim is to enable the efficient transduction of retinal endothelial cells, thus preventing the progression of the disease.

Objective

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.

Coordinator

THE PROVOST, FELLOWS, FOUNDATION SCHOLARS & THE OTHER MEMBERS OF BOARD, OF THE COLLEGE OF THE HOLY & UNDIVIDED TRINITY OF QUEEN ELIZABETH NEAR DUBLIN
Net EU contribution
€ 150 000,00
Address
College green trinity college
D02 CX56 Dublin 2
Ireland

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Region
Ireland Eastern and Midland Dublin
Activity type
Higher or Secondary Education Establishments
Links
Other funding
€ 0,00