Gene therapy cures child blindness

Gene therapy cures child blindness

The US researchers have been able to reverse the damaging effects of a rare inherited and progressive eye disease called Leber’s congenital amaurosis by using gene therapy.This genetic disease appears at birth, or in the first months of birth and affects around 1 in 80,000 of the population. The disease stricken child starts losing his/her sight at birth and is completely blind by age  of 40.
 
Children born with this disease, have defects in a gene called RPE65, have defects in a gene called RPE65 that helps the  retina’s light sensing cells make rhodopsin.This is a pigment of the retina that is responsible for both the formation of the  photoreceptor cells and the first events in the perception of light. Without rhodopsin, the photoreceptor cells gradually die,leading to blindness.

In a clinical trial, when gene RPE65 was injected into the retina of patients with LCA using a harmless virus as carrier, their eye sight showed marked improvement.