Gene Therapy Gives Sight to Blind Children With Rare Disorder

Eight-year-old Corey Haas stared skyward four days after he had been to the doctor and noticed for the first time in his life that the sun hurt his eyes.

He had undergone experimental gene therapy to correct an inherited defect that left him with little vision and likely to go blind as an adult. The experiment, described today in the journal Lancet, worked.

The youngster from Hadley, New York, was the seventh of 12 people with the condition known as Leber’s congenital amaurosis to have a gene injected into one eye. All of the patients improved. The success of the experiment is a major boost for the field of gene therapy, which began 20 years ago and has had few clear successes in that time, said Ronald Crystal, a gene therapy researcher who wasn’t involved in the study.

“This is the most successful gene therapy trial yet in terms of showing improvement in human disease,” said Crystal, a professor of medicine at Weill Cornell Medical College in New York. “I think the field is now turning around.”

A number of companies are developing gene therapies and 320 trials are under way or cleared to begin by U.S. regulators, said Karen Riley, a U.S. Food and Drug Administration spokeswoman. Genzyme Corp. of Cambridge, Massachusetts, will begin a human trial using gene therapy next year to treat macular degeneration, the leading form of age-related vision loss, said John Lacey, a Genzyme spokesman, in a telephone interview yesterday. ...

via Gene Therapy Gives Sight to Blind Children With Rare Disorder - Bloomberg.com.