Recently, scientists and doctors have achieved a striking result, overcoming one of the worst problems in medicine - congenital blindness in children. Due to the innovative genetic therapy conducted in London, children born with a rare genetic disease congenital Amavrosis Leber (LCA) managed to return their vision and return them to normal life.
Amavrosis of Leber is a serious genetic retinal disease caused by mutations in the AIPL1 gene. This disorder usually leads to irreversible blindness from birth. However, thanks to the latest achievements in genetic therapy, doctors were able to change this condition. The operation lasted only 60 minutes and allowed the children to see the outside world, begin to recognize the faces of parents, read and even write. The unique procedure was performed at the London Hospital Murfields, and the results exceeded all expectations. Four children, aged from one to two years, from different countries, such as the United States, Turkey and Tunisia, have received treatment that allowed them to restore their vision. Genetic therapy was to deliver a functional copy of the AIPL1 gene through a harmless virus that aims at the retina. This allows you to restore the work of photoreceptors, cells that are responsible for the perception of light and the transmission of visual information to the brain. The results of the operation were incredibly positive. One of the notable cases is the six -year -old Jace of Connecticut (USA), who could not follow the objects, even if they were kept very close to his face. After the operation, his vision improved so much that he was able to identify toys at a distance and even jokingly "steal phones" with his teachers.
These results confirm the great potential of genetic therapy in the treatment of severe childhood blindness, and open new prospects for early intervention and treatment of such diseases. Professor Michelle Michaelides, an ophthalmology consultant, called this breakthrough in medicine extremely impressive and emphasized the importance of further research in this area.