1. Gene therapy is the transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.

Two trials in gene therapy on Leber’s congenital amaurosis, an inherited form of blindness, have shown signs of temporary vision improvement, according to scientists. Laber’s congenital amaurosis is a childhood-onset autosomal recessive blindness, known to be caused by mutations in at least 19 different genes. This condition was considered to be untreatable until 2008, when gene therapy was successfully developed for patients with the disease caused by mutated RPE65 gene. The study published in the New England Journal of Medicine observed the effects of injecting healthy genes into the retinas of 12 young patients in a period of six years. They scientists found that fifty percent of patients showed signs of improvement in their night vision, peaking at six to twelve months after the treatment. In these patients, the treated retina showed improved visual sensitivity, which slowly increased in area and then contracted. Unfortunately their daytime vision did not improve. Gene therapy improves night vision but there is no evidence that it slows progression of the disorder. (…) We now need a more potent gene therapy vector. — Robin Ali Scientists from the University of Pennsylvania led another small trial and have found a similar pattern of improving eyesight lasting between one to three years after the treatment, according to BBC News.

Read more at: http://www.immortal.org/8218/gene-therapy-could-be-a-solution-for-inherited-blindness-study-suggests/
Source: Immortal News