Abstract

Optogenetics may enable mutation-independent, circuit-specific restoration of neuronal function in neurological diseases. Retinitis pigmentosa is a neurodegenerative eye disease where loss of photoreceptors can lead to complete blindness. In a blind patient, we combined intraocular injection of an adeno-associated viral vector encoding ChrimsonR with light stimulation via engineered 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. The patient perceived, located, counted and touched different objects using the vector-treated eye alone while wearing the goggles. During visual perception, multichannel electroencephalographic recordings revealed object-related activity above the visual cortex. The patient could not visually detect any objects before injection with or without the goggles or after injection without the goggles. This is the first reported case of partial functional recovery in a neurodegenerative disease after optogenetic therapy.

Extract:

Taken together, the psychophysical and neurophysiological evidence presented in this article suggest that the optogenetic stimulation of human retinal ganglion cells by a light-projection system linked to a camera is a promising way to partially restore vision in blind patients affected with advanced RP.

https://www.nature.com/articles/s41591-021-01351-4

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Optogenetic therapy, or manipulating proteins and cells with light, is an advanced technology developed in the early 2000s that drove major discoveries about the inner workings of our brains. Yet, while actively researched in experimental animals, functional improvement using this method was never reported in humans—until now.

In a paper published today in Nature Medicine, scientists from Paris, Pittsburgh and Basel, Switzerland, reported the first-ever case of partial vision recovery in a blind patient after optogenetic therapy. The pioneering study describes the first time a patient has achieved partial functional recovery in any neurodegenerative disease by using optogenetic tools.

“I hope it will be a major breakthrough,” said first and corresponding author, José-Alain Sahel, chair of ophthalmology at the University of Pittsburgh, director of the UPMC Eye Center and professor at Sorbonne University in France. “This paper is a culmination of more than 12 years of work, and I am very pleased to have contributed to this effort with Botond Roska in Basel and all my colleagues at Institut de la Vision in Paris.”

Watch the video and read the rest of the article at https://www.pittwire.pitt.edu/news/first-time-optogenetic-therapy-partially-restores-patient-s-vision

Read more about Sahel’s vision for curing blindness in Pitt Med magazine.

And read coverage of the new paper in The New York Times and BBC.

The post For the First Time, Optogenetic Therapy Partially Restores Patient’s Vision appeared first on EMFSA.