Optogenetic vision restoration 3, 4, 5 is a mutation-independent approach for restoring visual function at the late stages of RP after vision is lost 6, 7, 8, 9. With the exception of a gene replacement therapy for one form of early-onset RP caused by mutation in the gene RPE65 (ref. It affects more than 2 million people worldwide. Retinitis pigmentosa (RP) is a progressive, inherited, monogenic or rarely digenic 1 blinding disease caused by mutations in more than 71 different genes ( ). This is the first reported case of partial functional recovery in a neurodegenerative disease after optogenetic therapy.

The patient could not visually detect any objects before injection with or without the goggles or after injection without the goggles. During visual perception, multichannel electroencephalographic recordings revealed object-related activity above the visual cortex.

The patient perceived, located, counted and touched different objects using the vector-treated eye alone while wearing the goggles. The goggles detect local changes in light intensity and project corresponding light pulses onto the retina in real time to activate optogenetically transduced retinal ganglion cells. In a blind patient, we combined intraocular injection of an adeno-associated viral vector encoding ChrimsonR with light stimulation via engineered goggles. Retinitis pigmentosa is a neurodegenerative eye disease where loss of photoreceptors can lead to complete blindness. Optogenetics may enable mutation-independent, circuit-specific restoration of neuronal function in neurological diseases.

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