Scientists have for the first time successfully used gene therapy to partially restore the sight of patients with a rare form of blindness known as Leber’s congenital amaurosis. Some of the patients who were only able to detect hand motions were in a matter of weeks able read a couple lines on an eye chart. This blindness is caused by a mutation in the gene that is responsible for making the protein that is in turn needed by the retina. This protein is in charge of sensing light and sending images to the brain and people without this working gene gradually lose their sight until it is completely gone around early adulthood.
Gene therapy is basically the process of removing a faulty gene and replacing it with a normal one and over the years has had limited success. This study, however is a major breakthrough for gene therapy, as stated by some scientists “I think it’s a really big shot in the arm for gene therapy and for medicine in general,” said Dr Ronald Crystal, who is head of genetic medicine at Weill Cornell Medical College in New York. They achieved this breakthrough by injecting millions of copies of the healthy working gene behind the retina. And although not a huge improvement was seen in all, this is just the beginning and over time hopefully we will be able to perfect this technique to completely reverse blindness.
by Adam McNicol 41202544