Blindness - Research

Research

A 2008 study published in the New England Journal of Medicine tested the effect of using gene therapy to help restore the sight of patients with a rare form of inherited blindness, known as Leber's congenital amaurosis or LCA. Leber's Congenital Amaurosis damages the light receptors in the retina and usually begins affecting sight in early childhood, with worsening vision until complete blindness around the age of 30.

The study used a common cold virus to deliver a normal version of the gene called RPE65 directly into the eyes of affected patients. Remarkably all 3 patients aged 19, 22 and 25 responded well to the treatment and reported improved vision following the procedure. Due to the age of the patients and the degenerative nature of LCA the improvement of vision in gene therapy patients is encouraging for researchers. It is hoped that gene therapy may be even more effective in younger LCA patients who have experienced limited vision loss as well as in other blind or partially blind individuals.

Two experimental treatments for retinal problems include a cybernetic replacement and transplant of fetal retinal cells.


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