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Gene therapy for inherited blindness disorder improves eyesight in short term, study shows

BMJ 2015; 350 doi: https://doi.org/10.1136/bmj.h2376 (Published 05 May 2015) Cite this as: BMJ 2015;350:h2376
  1. Susan Mayor
  1. 1London

Gene therapy for an inherited disorder that causes vision loss in childhood improves eyesight for one to three years, but the benefits then diminish, two early phase studies have shown.

The researchers said that the studies, published in the New England Journal of Medicine,1 2 demonstrate the potential benefits and the remaining challenges of using gene therapy to treat retinal conditions.

The two studies treated people with Leber’s congenital amaurosis, a group of conditions that cause blindness early in life owing to mutations in at least 19 different …

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