Gene therapy transforms eyesight of 12 people with rare visual defect

Pennsylvania researchers using gene therapy have made significant improvements in vision in 12 patients with a rare inherited visual defect, a finding that suggests it may be possible to produce similar improvements in a much larger number of patients with retinitis pigmentosa and macular degeneration.

The team last year reported success with three adult patients, an achievement that was hailed as a major accomplishment for gene therapy. They have now treated an additional nine patients, including five children, and find that the best results are achieved in the youngest patients, whose defective retinal cells have not had time to die off.

The youngest patient, 9-year-old Corey Haas, was considered legally blind before the treatment began. He was confined largely to his house and driveway when playing, had immense difficulties in navigating an obstacle course and required special enlarging equipment for books and help in the classroom. Today, after a single injection of a gene-therapy product in one eye, he rides his bike around the neighborhood, needs no assistance in the classroom, navigates the obstacle course quickly and has even played his first game of softball.

The results are "astounding," said Stephen Rose, chief scientific officer of the Foundation Fighting Blindness, which supported the work but was not involved directly. "The big take-home message from this is that every individual in the group had improvement . . . and there were no safety issues at all."

http://www.latimes.com/news/nationworld/nation/la-sci-gene-therapy25-2009oct25,0,2334183.story

:applause: Video of cured 9 year old at link.

:sarcasm:

to be able to read some GOOD NEWS with Sunday AM coffee! Thanks for this post. Ms Bigmack

I've known at least one person with retinitis pigmentosa, maybe two. This is exciting news.