Leber’s hereditary optic neuropathy (LHON) is a rare genetic disease of the eye due to an abnormality of a mitochondrial gene which causes a sudden asymmetric loss of central vision, generally between 15 and 30 years of age. There is no treatment for this disease at present.
Gene therapy project
- Reference laboratory: Genethon
- Partner: Institut de la Vision
This project, which is at the preclinical stage today, is based on the administration of an AAV (Adeno Associated Virus) vector carrying a normal copy of the gene which is mutated in patients. It is being carried out in the context of a collaboration with Dr. Marisol Corral-Debrinski and Prof. Jose-Alain Sahel (Institut de Vision, Paris). An observational clinical study is in progress for a trial which is due to begin in 2013.
