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Gene therapy restores sight to two children

2019-12-16T11:44:11.122Z


Two small patients of eight and nine years suffering from a particular form of hereditary retinal dystrophy that made them visually impaired from birth recovered their sight thanks to an innovative technique performed for the first time in Italy in the Clinic ... (ANSA)


Two small patients of eight and nine years suffering from a particular form of hereditary retinal dystrophy that made them visually impaired from birth recovered their sight thanks to an innovative technique performed for the first time in Italy in the Eye Clinic of the University of Campania ' Luigi Vanvitelli 'in collaboration with Novartis. The disease is caused by mutations in a gene called RP65 and the therapy provides a functioning copy of this gene that can, through a single administration, improve vision.

The results obtained with the revolutionary gene therapy, called 'Luxturna', were illustrated by Francesca Simonelli, director of the Eye Clinic of the University Vanvitelli. The disease is caused by mutations in a gene called RP65 and the gene therapy called 'Luxturna' (Voretigene neparvovec) provides a functioning copy of this gene and is able, through a single administration, to improve patients' vision. The therapy was developed thanks to the collaboration between Novartis, the pharmaceutical company, and the Neapolitan University that was chosen to start treating the drug.

Source: ansa

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