after 10 years of study, the first gene therapy clinical trial finally began. Were close to the blind, or only light perception. Experts say early tests of the data exciting.
tests on a small scale, a total of three patients. But they are receiving treatment only a week later, visual acuity improved as magical. The results were published in National Academy of Sciences on the electronic version (PNASEarlyEdition)
There are also two test results have been published. Each test were only three patients, who only a very weak sense of light, access to genetic or congenital blindness. After treatment, the patient's visual acuity and vision have been improved.
These clinical trial showed that gene therapy and modern medicine has made great victory to have a situation similar to that of blind friends with great hope that these blind people in the past appears to be no way of treatment. famous U.S. John's. Ophthalmology professor at Johns Hopkins University Merton. F. Burger King said. He is not a test of the participants.
blind
gene therapy trials in patients suffering from a congenital amaurosis called Lieber's disease (Lebercongenitalamaurosis). Lieber
suffering from congenital amaurosis patients poor vision at birth, with age and the rapid growth of recession, when most of the adults who have been totally blind.
disease may be due to mutations in a gene therapy approach is
to patients with retinal cells into a normal, healthy copy of the gene, called RPE65 gene, the gene expression so that it may have lost the function of cells to re – Access to function. This requires special treatment or phage viruses as a gene carrier.
In the beginning, people worried that the major gene therapy is safe. But people soon found that the treatment can rapidly improve the patient's vision.
researchers in the paper the words elated to describe the response of subjects.
subjects are involved in the pilot when 20-year-old head Gangchu
participants are brave, they do not worry about security issues, in their view, to vision than anything else, but in the treatment 7 — 10 days later, their vision was improved, the researchers said, to improve the speed of vision is amazing. However
results can not be said to be perfect, because the patient's dark vision has not improved, compared with normal subjects dark adaptation of the apparent extension of the time. In the normal 10 minutes would adapting the dark about the environment, but these were spent 8 hours, or even a longer time to adapt.
This tells us that the future need to explore different concentrations of the carrier in order to improve the situation. The researchers said.
in the other two tests, one Subjects appeared retinal damage, but researchers believe that this is the complications caused by surgical implantation of gene and not caused by the injection of the gene.