Community Research and Development Information Service - CORDIS

FP7

Periodic Report - AAVEYE (GENE THERAPY FOR INHERITED SEVERE PHOTORECEPTOR DISEASES)

Project ID: 223445
Funded under: FP7-HEALTH

Abstract

The retina represents an ideal target for gene therapy approaches because of the size of the eye, which allows the use of small vector doses, and because of its immune-privileged environment. Furthermore, the presence of the blood–retinal barrier, the retinal pigmented epithelium (RPE) and the intracellular junction in the inner retina avoids vector spreading to the systemic circulation.

The efficiency of the therapy can be easily monitored via non-invasive and quantitative methods, such as electroretinography (ERG), ophthalmoscopy, optical coherence tomography, the measurement of afferent papillary responses and visual evoked potentials.

This report, which includes an image of the consortium logo summarises their achievements and notes that the results of this research project will hopefully lead to the development of strategies for the treatment of inherited retinal degenerations, in particular severe forms of LCA and RP.

Scientific discoveries from this work could have an immediate impact on patients health as preclinical data obtained in animal models could be the starting point for the translation into clinical applications in humans, in agreement with the proposed bench-to-bedside approach.

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Related information

Record Number: 11329 / Last updated on: 2011-08-02
Category: PROJ