Gene therapy saved from blindness

BBC news: Gene therapy reverses sight loss and is long-lasting

Scientists have tested gene therapy on patients with choroideremia, a progressive degradation of the retina in men that leads to blindness. The new method, consisting in the introduction of a working copy of the damaged gene, which caused the development of the disease, was tested on 14 patients in the UK and another 18 patients from the USA, Canada and Germany. The experiments have been conducted over the past four and a half years.

Robert Maclaren explains that gene therapy not only helped to stop the progression of the disease, but also allowed to restore already damaged vision. In some cases, the improvements were significant. He also notes that a single injection is usually required, which allows correcting a gene defect.

In the event that the next stages of research, in which a much larger number of patients will participate, will be successful, gene therapy for the treatment of choroideremia will be allowed for clinical use in three years.

In the near future, the same team of scientists from Oxford University (OxfordUniversity) plans to treat other diseases that also lead to blindness in the same way, including macular degeneration and retinal pigment dystrophy. However, it will be more difficult to cope with them, the authors note, since their occurrence is not due to damage to not one gene, but several.

Portal "Eternal youth" http://vechnayamolodost.ru  10.05.2016