Nature Medicine: blind patient partially regains sight after GS030 optogenetic therapy

GenSight Biologics/GS030 optogenetic therapy

A team led by José-Alain Sahel and Botond Roska reported that a man blind from retinitis pigmentosa could perceive, locate and count objects with his treated eye after GS030, when wearing the goggles. It is the first reported partial functional recovery in a neurodegenerative disease after optogenetic therapy.

• Gene for ChrimsonR, which responds to amber light, delivered to the eye by an AAV vector
• Goggles with a camera project the scene onto the retina as amber light
• Without the goggles the patient could not complete the tests
• Roska: proof of concept; Sahel: it will take time before patients can be offered it