- The Washington Times - Monday, April 28, 2008

NEW YORK (AP) — Scientists for the first time have used gene therapy to dramatically improve sight in people with a rare form of blindness — a development researchers called a major advance for the experimental technique.

Some vision was restored in four of the six young people who received the treatment, teams of researchers in the United States and Britain reported yesterday. Two of the volunteers who could see only hand motions were able to read a few lines of an eye chart within weeks.

“It’s a phenomenal breakthrough,” said Stephen Rose, chief research officer of the Foundation Fighting Blindness, which helped pay for one study conducted at Children’s Hospital of Philadelphia.

If successful in larger numbers, researchers said, the technique has the potential to reverse blindness from other kinds of inherited eye diseases.

“I think this is incredibly exciting,” said Dr. Jean Bennett, a professor of ophthalmology at the University of Pennsylvania and a leader of the Philadelphia study. “It’s the beginning of a whole new phase of studies.”

The research was published online yesterday by the New England Journal of Medicine in conjunction with presentations at a medical meeting in Florida.

The two teams of scientists, working separately, each tested gene replacement therapy in three patients with a form of a rare hereditary eye disease called Leber’s congenital amaurosis. No treatment is available for the disease, which appears early in infancy and causes severe vision loss, especially at night.

An estimated 2,000 Americans have the form of the disease they targeted, Dr. Bennett said.

Gene therapy — replacing faulty genes with a normal version — has been studied in humans for more than 15 years with limited success. The field suffered a setback with the 1999 death of Jesse Gelsinger, 18, in an experiment for a liver disorder at Penn. Some children treated for an immune disorder called the “bubble boy disease” later developed leukemia.

The early results of the eye experiments should give the field a boost, some researchers said.

“I think it’s really a big shot in the arm for gene therapy and for medicine in general,” said Dr. Ronald Crystal, head of genetic medicine at Weill Cornell Medical College in New York.

Each of the study participants had mutations in a gene that makes a protein needed by the retina, which senses light and sends images to the brain. Those without the gene gradually lose sight until they are blind in early adulthood.

The retina itself stays in relatively good shape for a while, making it a good candidate for gene therapy, said Robin Ali, a professor at University College London, who led the British team. He likened the defective gene to a missing spark plug in a car engine.

“The whole engine can be absolutely fine, but if it doesn’t have a spark plug, the car’s not going to work,” Mr. Ali said.


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