The BBC reports - an international group of researchers found that experimental gene therapy is effective in combating a hereditary disease that leads to blindness, according to Pannochka, an online publication for girls and women aged 14 to 35.. net The first stage of the study was conducted with a group of 12 patients with Leber's congenital amaurosis, which is a genetic disease of the retina.

In this disease, the death of photosensitive cells occurs, leading to a sharp deterioration in vision almost to complete blindness, as well as to nystagmus and the absence of pupil reaction to light.

Each of the patients participating in the study, whose vision was practically absent, was introduced into one eye a viral vector containing the RPE65 gene. A defect in this gene underlies the disease..

According to the results of the first stage, in 2008, it was found that such experimental therapy slightly improved the eyesight of the subjects without particularly pronounced side effects.

In the next phase of the study, the gene preparation AAV2-hRPE65v2 was injected into the other eye in three participants.

Which led to a subsequent improvement in vision and patients could already see at dusk, make purchases in the store and recognize people's faces.

The results of magnetic resonance imaging, which was performed by volunteers, showed that the visual centers of the brain, when examining various objects, were activated, said Manzar Ashtari from the Philadelphia Children's Hospital.

The remaining nine patients, scientists intend to conduct second eye therapy, said study leader Gene Bennett.

medbe. ru.