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Therapy may yet stem from cells in the retina
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  1. R R Ali1,2,
  2. J C Sowden2
  1. 1Institute of Ophthalmology, University College London, Bath Street, London EC1V 9EL, UK
  2. 2Institute of Child Health, University College London, Guilford Street, London WC1N 1EH, UK
  1. Correspondence to: Robin R Ali, Institute of Ophthalmology, University College London, Bath Street, London EC1V 9EL, UK; r.ali{at}ucl.ac.uk

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The eye is outstandingly well suited for the development of new treatments

The paucity of effective treatments for inherited retinal disease and age related macular degeneration (AMD) underlines the need for the development of novel therapeutic approaches such as gene therapy. Luckily, the eye is outstandingly well suited for the development of new treatments. Non-invasive procedures for the determination of ocular structure and function in both the clinic and laboratory are better developed than for any other organ. Ease of access to the eye enables manipulation at all levels from subcellular to whole organ; the understanding of the eye, from developmental biology to biochemistry, is highly advanced and there are many established animal models of retinal disease. This has enabled rapid progress in the development of gene therapy approaches for retinal disease compared with that for other forms of neurological disorders. While there has been tremendous progress over the past 10 years with the development of gene therapy strategies, this approach will not be effective once degeneration has occurred and some form of cell transplantation may be required to enable the replacement of lost photoreceptor cells. The first trials of cell replacement have been performed using mature photoreceptors or their late progenitors …

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