United States: Scientists have already found a way to help people who are born with a rare eye disease which is called Leber congenital amaurosis. This specific disease makes people lose their sight early in life because of problems with a gene called GUCY2D, which helps us see.
Researchers and the health experts are using gene therapy to fix this gene and restore vision. This could be great news for the cute little kids and adults who have lost their sight due to this condition. About 100,000 people around the world have this illness.
Side Effects and Ongoing Research
As reported by HealthDay, Side effects could happen and were usually related to the survey which is used to implant the gene therapy which include tiny hemorrhages within the eye that later healed or the transient inflammation of the eye treated with the steroids.
The patients vision is being is being tracked or going forward the researchers said and the bigger randomized trial is being planned as a requirement of any US food and the drug administration approval of the new therapy.
Building on Previous Success
The new work also follows on the team’s prior success using gene therapy to reverse another form of the Leber congenital amaurosis and this time is connected to a mutation in the CEP290 gene.
So this particular treatment success in our most recent clinical trials together with our earlier experience beings hope for a viable treatment for about 20 percent of infantile blindness caused by the inherited retinal degenerations and the study and the co-author and the Dr. Tomas Aleman said in a UPenn news release.
He is research professor in the ophthalmology and the co-director of the Center for Hereditary Retinal Degeneration.
“The focus now is on perfecting the treatments and treating earlier manifestations of these conditions once safety is confirmed,” he said. “We hope similar approaches will lead to equally positive outcomes in other forms of congenital retinal blindness.”