Gene Therapy Cures Blindness

Reported December 2010

GAINESVILLE, FL (Ivanhoe Newswire) -- There are more than 15 hundred gene therapy trials going on around the world to treat everything from Parkinson's to heart disease. But one of the most successful experiments comes from a gene therapy trial to treat blindness.

A workout on wheels was something Dale Turner thought was impossible.

"This is like destiny, this is like a miracle that I never expected," Dale Turner, gene therapy patient, told Ivanhoe.

He was born with an inherited form of blindness called Leber's congenital amaurosis. It's a defective gene that prevents his retina from producing an enzyme essential for vision.

"The doctors said I would be completely blind by the age of 10, " Turner said. "You always have the hope that there will be something for your incurable condition growing up. There'll be something for my eyes someday."

That something came in the form of gene therapy. In a clinical trial, ophthalmologists injected billions of copies of a new, healthy gene beneath Dale's retina.

"When it reaches the cell level, it actually can insert into the DNA sequence of the individual and replace the bad sequence with a normal sequence," William Driebe, M.D., an ophthalmologist at the University of Florida, explained.

These genes stimulate the retina to produce the missing enzyme. 12 people have received the treatment, all saw improvement, and 50 percent are no longer classified as legally blind. Results have lasted more than three years.

"They've all experienced increased vision just by gene therapy, replacing a defective gene with a normal gene so this is really just startling," Dr. Driebe said.

Dale's condition is called a single point mutation, now ophthalmologists are studying how to use gene therapy to treat more common diseases with multiple mutations, including macular degeneration, diabetic retinopathy and glaucoma.

"I think this is the cutting edge for ophthalmic research right now," Dr. Driebe said.

Dale will never forget his first step outside after surgery.

"I could see colors like never before and it was just like I saw a blue sky like I've never seen before," Turner said. "I just had this feeling like what have I been missing out this whole time? My whole life."

He's now studying to be a lawyer and hopes the treatment he calls 'His Miracle' helps others see the world. Researchers exercised caution by treating only one eye, but new findings suggest that patients may experience similar benefits from the gene therapy in the other eye.

Click here to Go Inside This Science or contact:

William Driebe, MD
Professor and Chair of Ophthalmology
University of Florida
Gainesville, FL
bdriebe@eye.ufl.edu

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