¹Division of Molecular Therapy, UCL Institute of Ophthalmology and UCL/Moorfields Eye Hospital Biomedical Research Centre for Ophthalmology, London, UK

Correspondence: Professor RR Ali, Division of Molecular Therapy, UCL Institute of Ophthalmology and UCL/Moorfields Eye Hospital Biomedical Research Centre for Ophthalmology, London EC1V 9EL, UK. E-mail: r.ali@ucl.ac.uk

Received 27 February 2008; Revised 5 March 2008; Accepted 6 March 2008; Published online 17 April 2008.

Abstract

A wide range of retinal disorders can potentially be treated using viral vector-mediated gene therapy. The most widely used vectors for ocular gene delivery are based on adeno-associated virus (AAV), because they elicit minimal immune responses and mediate long-term transgene expression in a variety of retinal cell types. Proof-of-concept experiments have demonstrated the efficacy of AAV-mediated transgene delivery in a number of animal models of inherited and acquired retinal disorders. Following extensive preclinical evaluation in large animal models, gene therapy for one form of inherited retinal degeneration due to RPE65 deficiency is now being tested in three concurrent clinical trials. Here, we review different approaches for treating inherited retinal degenerations and more common acquired retinal disorders using AAV-based vectors.