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Gene therapy improves sight

By Tim Sandle     Jan 18, 2014 in Science
Following a gene therapy trial, patients who were progressing toward blindness now have better vision after completing the treatment.
After completing gene therapy, patients with a rare degenerative eye disease called choroideremia had partially-restored vision. Choroideremia is a retinal degenerative disease that leads to the degeneration of the choriocapillaris, the retinal pigment epithelium, and the photoreceptor of the eye. The condition is a rare inherited disorder that only occurs in males.
For the trial, researchers used a genetically modified virus and inserted it into the cells in the retina of the eye without causing damage, according to International Business Times. . The research team had to detach the patients’ retinas to inject the rescue gene.
Gene therapy
is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease.
After the trial, two patients with poor vision saw improvements after the trial, while those with better vision did not experience any declines. Toby Stroh, one of the participants in the trial, told The Guardian that "his result does not make me swing from the chandeliers. I refuse to say everything is going to be roses. But there is hope."
The results of the trial have been published in The Lancet, in a paper titled "Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial."
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