Yeah, just put those in my eye. Thanks.
Six percent of people with LCA-caused blindness have a defective RPE65 gene. It just so happens you can insert a functional copy of the gene into a particular type of virus. Then what? Well, if you’re these guys, you inject up to 150 billion of these virus cells into one eye of a dozen sight-impaired kids. Sure, why not? Mwahahahaha! *thunder clap, ominous organ music*
All can now see better in dim light, have an increased visual field, their pupils respond to bright light. Seven of the 12 have increased visual acuity.
And all of the kids involved. . . None of them could navigate a small obstacle course scientists set up for them pre-treatment. One video is this cute little boy, nervously twisting his shirt, and just standing there. He was afraid to take one step on the course. He couldnt see anything.
Yeah, after treatment, all the kids could navigate it. That scared kid? Brazenly flies through the course in a minute. No hesitation. And acting like a dopey 10 year old boy. [scienceblogs]
Wait, it worked? I thought we were just placing bets on how many people would let somebody in a white coat spray viruses all up in their eye. You know, for kids!