A remarkable story of sight regained has been reported by Science Translational Medicine magazine. Gene therapy has been used to treat Leber’s Congenital Amaurosis (LCA), an inherited disease that attacks eyesight at an early age. The retina is prevented from functioning normally, leading to severely impaired vision, involuntary eye movements and extremely poor night vision. Vision is affected to the point that spectacles and contact lenses do not improve
condition.
However, three US citizens who have the disease reported a dramatic improvement in their vision after having an injection of an engineered virus carrying the gene RPE65. One of the patients, Tami Moorehouse described the joy in seeing her children’s faces after her second eye was injected. “Life is so much easier at a level that most people take for granted. Any amount of vision that you can get when you have almost nothing is incredibly valuable”, she said.
Professor Robin Ali of the Institute of Ophthalmology at University College London heads a UK team carrying out similar gene therapy trials. His opinion: “This is confirmation that it is possible to administer gene therapy safely to the second eye of patients. This is reassuring and increases the prospect of this type of therapy for treatment of a wide range of eye conditions.”
BBC reported the STM’s findings on February 8th.
