ABSTRACT The eye has unique advantages as a target organ for gene therapy of both inherited and acquired ocular disorders and offers a valuable model system for gene therapy. The eye is

readily accessible to phenotypic examination and investigation of therapeutic effects _in vivo_ by fundus imaging and electrophysiological techniques. Considerable progress has been made in

the development of gene replacement therapies for retinal degenerations resulting from gene defects in photoreceptor cells (rds, RPGRIP, RS-1) and in retinal pigment epithelial cells (MerTK,

RPE65, OA1) using recombinant adeno-associated virus and lentivirus-based vectors. Gene therapy also offers a potentially powerful approach to the treatment of complex acquired disorders

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_Invest Ophthalmol Vis Sci_ 2004; 45: 2194–2200. Article  Google Scholar  Download references ACKNOWLEDGEMENTS James Bainbridge is a Wellcome Advanced Fellow (Grant 074617/Z/04/Z); Mei Hong

Tan is supported by the UK Department of Health. AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * Division of Molecular Therapy, Institute of Ophthalmology, University College London, London,

UK J W B Bainbridge, M H Tan & R R Ali Authors * J W B Bainbridge View author publications You can also search for this author inPubMed Google Scholar * M H Tan View author publications

You can also search for this author inPubMed Google Scholar * R R Ali View author publications You can also search for this author inPubMed Google Scholar CORRESPONDING AUTHOR Correspondence

to R R Ali. RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Bainbridge, J., Tan, M. & Ali, R. Gene therapy progress and prospects: the eye. _Gene

Ther_ 13, 1191–1197 (2006). https://doi.org/10.1038/sj.gt.3302812 Download citation * Received: 03 January 2006 * Revised: 05 May 2006 * Accepted: 19 May 2006 * Published: 13 July 2006 *

Issue Date: 01 August 2006 * DOI: https://doi.org/10.1038/sj.gt.3302812 SHARE THIS ARTICLE Anyone you share the following link with will be able to read this content: Get shareable link

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lentivirus * retina * RPE65