Ocular Gene Therapy
- 1 November 1993
- journal article
- research article
- Published by American Medical Association (AMA) in Archives of Ophthalmology (1950)
- Vol. 111 (11) , 1477-1479
- https://doi.org/10.1001/archopht.1993.01090110043019
Abstract
THIS ISSUE of theArchivescommemorates the 40th anniversary of the publication of Watson and Crick's1classic paper on the structure of DNA. In 1953, gene therapy was mentioned only in the realm of science fiction. Now, a mere 40 years later, gene therapy has become a reality.2A number of clinical trials are already in progress, and the number of proposed trials is increasing almost daily.2-5Current trials involve diseases as diverse as severe combined immunodeficiency, cancer, and cystic fibrosis. Although to date none of these trials involves the eye, this situation is sure to change in the near future. (In one sense, ocular gene therapy is already a reality. The murineretinal degeneration[rd] andretinal degeneration slow[rds] mutations have been "rescued" using transgenic mouse technology.6,7However, transgenesis involves germ line transformation and is not directly applicable to human gene therapy.) Genetic diseaseKeywords
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