The first clinical trial to treat the Leber congenital amaurosis, an inherited disease that affects the retina causing blindness to children who suffer from the moment of birth. The disease in question affects the retina and specifically the photosensitive cells that capture the light thanks to retinol (fundamental substance in vision).
Until now there was no treatment against this disease, but after years of research, there is a hope of cure thanks to the development of various gene therapies by the scientific team belonging to the Institute of Ophthalmology at University College London and Moorfields Eye Hospital, both centers in the United Kingdom. The treatment involves the introduction of healthy RPE65 genes into the cells so that they start producing retinol. This substance is not produced with the disease because the genes responsible for production mutate. If everything goes as planned, healthy genes will begin their work and the functions of photosensitive cells will be restored. The researchers have an advantage, the treatment was applied in previous studies (with dogs), obtaining very satisfactory results.
Although you have to wait a few months to hear the verdict, a good result is predicted, every time you can cross out a disease from the list of incurable diseases, it is an achievement for science and humanity.
