Leber’s Hereditary Optic Neuropathy is a rare genetic disorder of the retina caused by a defect in a mitochondrial gene which causes a sudden and asymmetric loss of central vision, usually between the age of 15 and 30. There is currently no effective treatment for this condition.
This project, currently in the preclinical stage, is based on the administration into the patient of an AAV (adeno-associated virus) vector carrying a normal copy of the mutated gene. It is being conducted as part of a collaborative project with Dr Marisol Corral-Debrinski and Prof. José-Alain Sahel (Institut de la Vision, Paris).