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November 1993

Ocular Gene Therapy: From Fantasy to Foreseeable Reality

Author Affiliations

Baltimore, Md

Arch Ophthalmol. 1993;111(11):1477-1479. doi:10.1001/archopht.1993.01090110043019

THIS ISSUE of the Archives commemorates the 40th anniversary of the publication of Watson and Crick's1 classic 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.2 A number of clinical trials are already in progress, and the number of proposed trials is increasing almost daily.2-5 Current 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 murine retinal degeneration [rd] and retinal degeneration slow [rds] mutations have been "rescued" using transgenic mouse technology.6,7 However, transgenesis involves germ line transformation and is not directly applicable to human gene therapy.)

Genetic disease

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