New Gene Therapy Treatment for Blindness

There may be hope for people born with blindness according to researcher Robin Ali. The University of London's Institute of Opthalmology is testing a gene therapy treatment that would mitigate the affects of childhood blindness Clinical trials are beginning in Britain to test the safety of the treatment.

"We have been developing gene therapy for eye disease for almost 15 years but until now we have been evaluating the technology only in the laboratory," says Ali. "Testing it for the first time in patients is very important and exciting, and represents a huge step towards establishing gene therapy for the treatment of many different eye conditions."

Subjects in the testing are blind due to a rare condition called Leber's congenital amaurosis, which 1 in 80,000 Britons have. Children with the condition are born with a defective gene that eventually causes them to go blind by their 20's. If successful, the process could be modified to treat over 100 other types of inherited blindness.

The treatment requires for participants to be injected with a virus, which researches have altered to carry a corrected version of the defective gene. When the virus makes it to the eye, the degeneration happening in the cells of the retina will cease.

Safety is one of the most important aspects of the trial, and tumors are an especially dangerous risk. Tumors are unusual growths of tissue, caused by the mutation of DNA cells, which are cells that gene therapy works dangerously close with. A mutation that accidental activates an oncogene or reverses a tumor suppressor gene, may lead to a tumor. In 2003 a gene therapy trial went awry. Two children with severe combined i,a rare immune disease, developed leukemia during testing. The modified virus in that study produced cancerous tumors.

So far, results from tests on blind animals have been optimistic. "If this trial is successful it paves the way for the application of this technology in the treatment of many forms of inherited retinal degeneration and perhaps some of the more common disorders of the eye too," said Robin Ali

The teams leading retinal specialist, Tony Moore, added "It will be many months before we have the full picture. We anticipate the best outcome in younger patients, as we will be treating the disease in the early stages of its development"

Scientists are optimistic that a successful trial will eventually lead to gene therapy research for other more complex organs.
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