Saturday science subject: Gene therapy and blindness

— 5:43 PM on October 24, 2009

Can gene therapy help blind kids regain their sight? Yes, according to a new paper by researchers at the University of Pennsylvania and in the United Kingdom. As ScienceNow reports, the paper shows results of treatment for Leber's congenital amaurosis (LCA) in four children aged 8 to 11 as well as three adults.

A genetic defect, LCA causes the gradual death of photoreceptor cells in the retina, and it reportedly affects "about 3000 people" in the U.S. ScienceNow says the teams in the U.S. and Britain treated subjects using a modified virus carrying a "good copy" of the RPE65 gene, which is normally defective in LCA sufferers. The treatment was more effective in the children, improving light sensitivity in their retinas by up to four orders of magnitude:

One patient, Corey Haas, appears in a video 3 months after the treatment breezing through the obstacle course, following arrows and avoiding objects that he cannot see with his treated eye covered. Corey, 9, told reporters at a press conference this week that for the first time he can recognize faces, play baseball, read large print books, and ride his bike around his neighborhood alone.

You can watch videos of Corey's obstacle course run with and without treatment on the ScienceNow article page. The difference literally seems like night and day.

Study leader Jean Bennett says these trials are "an incredible launching pad to be able to target other diseases." ScienceNow quotes another researcher, Frans Cremers of Radboud University Nijmegen Medical Center in the Netherlands, as saying eight other genetic vision defects "have now been treated in mice and are ready to be tested in people."

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