Project description DEENESFRITPL 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. Show the project objective Hide the project objective 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. Fields of science medical and health sciencesmedical biotechnologygenetic engineeringgene therapynatural sciencesbiological sciencesmicrobiologyvirologymedical and health sciencesclinical medicineophthalmologymedical and health sciencesbasic medicinepathology Programme(s) HORIZON.1.1 - European Research Council (ERC) Main Programme Topic(s) ERC-2022-POC1 - ERC PROOF OF CONCEPT GRANTS1 Call for proposal ERC-2022-POC1 See other projects for this call Funding Scheme ERC-POC - Proof of Concept Grant 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 See on map Region Ireland Eastern and Midland Dublin Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00
