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Gene therapy for retinopathy of prematurity: the eye is a window to the future
  1. WILLIAM V GOOD
  1. Smith-Kettlewell Eye Research Institute, San Francisco, California, USA
  2. Cincinnati Childrens Hospital, Cincinnati, Ohio, USA
  1. ROBERT L GENDRON
  1. Smith-Kettlewell Eye Research Institute, San Francisco, California, USA
  2. Cincinnati Childrens Hospital, Cincinnati, Ohio, USA
  1. William V Good, MD, Smith-Kettlewell Eye Research Institute, 2318 Filmore Strett, San Francisco, CA 94115, USA Good{at}ski.org

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The eye is the window to the brain, the soul, and now perhaps even the future, with the exciting publication by Chowerset al in this month's issue of theBJO (p 991). The authors give us a view of the future as they elegantly demonstrate that gene transfer into blood vessels is possible in a rat model of retinopathy of prematurity (ROP). The future may be close at hand, because the eye once again offers an ideal organ to study the effects of this new therapy. ROP, a developmental disease of retinal angiogenesis in premature infants, is also an optimal and important disease to study potential therapeutic effects of gene therapy.

To date, the view of future gene therapy has been through hazy media at best. Despite incredible advances in developmental biology and molecular genetics during the past several decades, efforts to utilise these advances, and to deliver therapeutic genes to target organs or structures, have often been disappointing. The reason for disappointment usually hinges on delivery and expression of the therapeutic gene to the desired target. A vector must be used, one that transports a gene or genes to the target. The vector should be innocuous, sustainable, and, of course, able to deliver a genetic sequence that can be inserted …

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