Community Research and Development Information Service - CORDIS


Periodic Report - AAVEYE (Gene therapy for inherited severe photoreceptor diseases)

Project ID: 223445
Funded under: FP7-HEALTH


The ultimate aim of the AAVEYE project was to develop gene therapy strategies for the treatment of photoreceptors (PR) and Leber congenital amaurosis (LCA). This would be attempted through: 1. development of Adeno-associated virus (AAV)-based long-term and safe gene therapy to PR by combining endogenous promoters and AAV serotypes (WP1); 2. assessment of the impact of AAV-mediated photoreceptor transduction on rescue of visual function in animal models of severe RP and LCA (WP2); 3. evaluation of the efficacy of combination of gene replacement with adjuvant molecules on photoreceptor survival (WP3); and 4. characterisation of patients with inherited severe photoreceptor diseases to move the gene therapy strategies tested from bench to bedside (WP4).

The report includes an image of the project logo.

Download application/zip (1450324)

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