Gene therapy improves eyesight in LCA

Experimental gene therapy has shown promising results in improving eyesight in patients suffering from a rare eye disease known as Leber congenital amaurosis.

LCA is an inherited retinal degenerative disease characterized by severe loss of vision at birth, which may lead to total blindness by the age of 40.

The condition is associated with other eye-related abnormalities such as roving eye movements (nystagmus), deep-set eyes and sensitivity to bright light.

There is no effective treatment for the condition.

According to a report presented at the joint meeting of the American Academy of Ophthalmology and Pan-American Association of Ophthalmology in San Francisco, simple gene therapy improved eyesight in twelve LCA patients.

"All 12 patients given gene therapy in one eye showed improvement in retinal function," said lead researcher Katherine High, adding that it did not restore normal eyesight in either one of the patients.

In this method, an adeno-associated virus is adopted to carry the corrective gene called RPE65 -- a mutated variant of the gene is found in some 16 percent of LCA patients -- directly into the eyes, restoring the light-sensitive pigments in the retina.

The report to be published in The Lancet stated that the younger the patient is when treated with the therapy, the better the effects would be.

It further states that 'the efficacy will be improved if treatment is applied before retinal degeneration has progressed'.

Scientists hope their findings will not only pave the way for treating LCA patients but also for those with more common retinal diseases, such as age-related macular degeneration.

Source: PressTV.
Link: http://www.presstv.ir/detail.aspx?id=109584§ionid=3510210.