An international research team has shown that optogenetic gene therapy can help restore some vision in a patient blinded by hereditary retinitis pigmentosa. This is a milestone on the road to gene therapies for the potential reversal of blindness.
Clinical data published in Nature Medicine show for the first time that optogenetic methods can restore some vision to a blind person. This success is an important milestone on the way to developing gene therapies for hereditary diseases of light receptor cells - regardless of the mutations that cause these hereditary diseases. The data was published in Nature Medicine. They come from a study conducted by an international research team led by José-Alain Sahel and Botond Roska. The research team includes members of the Institut de la Vision and Hôpital National des Quinze-Vingts in Paris, the University of Pittsburgh, the Institute of Molecular and Clinical Ophthalmology Basel (IOB), StreetLab and GenSight Biologics (Euronext: SIGHT).
Optogenetics involves genetically modifying cells so that they produce light-sensitive proteins known as channelrhodopsins. This technique has been around for almost 20 years in the field of neuroscience, but until now the clinical utility of optogenetics has not been proven. The results reported today reflect 13 years of multidisciplinary efforts and are the culmination of the collaboration between the teams of José-Alain Sahel and Botond Roska.
Original publication: Sahel, JA., Boulanger-Scemama, E., Pagot, C. et al. Partial recovery of visual function in a blind patient after optogenetic therapy. Nat Med (2021).
