Tami Morehouse with functional blindness, which lasted for years, managed to partially restore vision by gene therapy, it is nothing but a miracle.Morehouse was the first of 12 patients with a rare congenital disease of the retina that three years ago, underwent an experimental treatment in one eye, and is one of three patients treated with gene therapy in both eyes.Morehouse 47 year old social worker and mother of three children before therapy was worried that it would lose even the vision that she had."My world just to decorate, and I could see things much more clearly.Shortly after the second session I went to have dinner with her husband and sitting and looking down, I thought: "Oh my god, I see the glass in front of me!".This treatment literally gave me a bright future ", - she says.
Gene therapy works on gene mutations eyes
study that Morehouse attended one of the first was conducted in order to show that gene therapy can improve vision in people with hereditary blindness."The new results show
Patients received injections of normal copies of the gene that functioned properly to the cells began to work better.Injections work so well that Bennett and his colleagues plan to conduct treatment of the second eye in the remaining five children and four adults who took part in the first study.
«There were some concerns that the first injection will induce an immune response, resulting in the body will reject the second injection," - says Bennett - "If this happens, the effect is achieved on the first eye could be jeopardized.But this did not happen. "After injection introduced in the second eye, three patients were able to see better in dim light, and two of the three were able to avoid obstacles in low light conditions.
Half of the patients got rid of blindness
All patients suffered from a hereditary degenerative disease of the retina, which is called Leber's congenital blindness caused by RPE65 mutation in the gene and the disease usually progresses to total blindness by mid-adulthood.
Morehouse says that her eyesight was getting worse with each of the three pregnancies.By the time she turned 40, she could distinguish a little light and colors, and most of the objects looked like "a dark, hazy, indistinct spots."Treatment constituted an injection of genetically modified virus carrying a healthy version of the gene RPE65.
After the first injection, seeing six patients out of 12 participants improved so that the diagnosis of blindness they no longer stood."A child of the participants in the original study had over one year to engage in cycling," - said study co-author Manazar Ashtar, Ph.D., Children's Hospital of Philadelphia."This child used a stick and held on to the adults to control the bike before they get treatment."
Gene therapy can prevent blindness
It is hoped that this therapy is focused on other mutations can be used to treat many other hereditary diseases that cause blindness.Katherine A. High, MD, Children's Hospital of Philadelphia, who also worked on the study, said, - "Now there are about 200 known genetic mutations that cause complete loss of sight."
Since Leber congenital blindness is a degenerative disease, it is hoped that the treatment can be used on very young children and babies even before until there was a loss of vision."In many genetic diseases, not only in blindness, early intervention may be critical for optimal results" - says Hai.
Morehouse very encouraged by this promise."If this treatment or therapy of this kind can help children keep the vision in the first place and save them from the struggle with the disease, it is truly a miracle," - she says.