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Gene Therapy Human Trial Will Inject Virus Into the Retinas of the Legally Blind

Accepted submission by takyon at 2016-02-29 19:23:19
Science

An upcoming human trial will attempt to use optogenetics [singularityhub.com] to treat conditions such as retinitis pigmentosa:

In the next month, scientists from RetroSense Therapeutics [retro-sense.com] will inject a virus deep into the retina [clinicaltrials.gov] of legally blind human volunteers. The virus will carry what is perhaps the most monumental payload in modern neuroscience history: DNA that codes for channelrhodopsin-2, a light-responsive protein isolated from algae that — under blue light — activates cells in the retina, thereby transmitting visual information to the brain.

Forget electronic implants [singularityhub.com]. If all goes well, these volunteers will be able to see again using their own eyes — but in no way a human being has ever experienced sight before. Whoa.

But the stakes are even higher: if this works, it means that optogenetics — a revolutionary neuroscience technique using channelrhodopsin-2 and other light-activated proteins — is feasible in humans as therapy. Considering optogenetics has been used in mice to implant false memories [sciencemag.org], treat cocaine addiction [popsci.com], attenuate OCD symptoms [nature.com], trigger sexual advances and aggression [nih.gov] and reverse motor deficits [stanford.edu] in Parkinson's disease — just to name a few feats— the technique could completely transform the face of neurology. "This is going to be a gold mine of information about doing optogenetics studies in humans," said [technologyreview.com] Dr. Antonello Bonci, the scientific director of the intramural research program at the National Institute on Drug Abuse, to MIT Technology Review.

[...] If it works, what will the patients see? No one can say for sure. After all, this will be the first time humans experience the visual world through the light sensor of algae. But studies with blind lab mice may give us a hint. In one previous study [nature.com], after optogenetics treatment, previously blind mice could swim out of a chamber in which the escape route was brightly lit. On average, they escaped as fast as mice with normal vision.


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