A boy and his little sister, aged 8 and 3, suffering from the same form of hereditary retinal dystrophy, have regained important visual skills following treatment with gene therapy. Today they better distinguish details and manage to move with confidence in poorly lit environments, without fear of tripping over objects. The interventions were carried out in collaboration by the ophthalmology units of the Agostino Gemelli IRCCS University Polyclinic Foundation and the Bambino Gesù Pediatric Hospital, as part of a project launched in 2021 for the common management of pediatric patients and adults suffering from hereditary retinal degenerations. The girl is the youngest patient in Italy to have received this treatment. The therapy consists of a single injection - “one shot” – into the sub-retinic space of both eyes of a working copy of the RPE65 gene. The healthy gene is carried within the cells by an associated adenovirus, with modified genetic heritage, which acts as a vector. Once in the cells, the working copy of the gene is able to restore the patient's visual ability in a meaningful and lasting way.