Special Reports

Gene Therapy Improves Eyesight In Patients With Vision Loss

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A new innovative procedure may be able to treat a rare form of progressive blindness, Fox News reported.

Surgeons at Oxford University treated six patients with choroideremia with a gene therapy technique that can hinder or slow down the visual degeneration caused by the disease. Choroideremia is a disease that causes the light-detecting cells at the back of the eye gradually dying, BBC News reported.

"Gene therapy is exciting; it's a new type of medicine," lead author Robert MacLaren, a professor at the University of Oxford, told Fox News.  "And what we're doing is it on a very small scale, because we're looking at a very straightforward gene to replace."

The therapy helped restore some of the sight patients have lost over the years. The treatment involves injecting the eye of the patient with a "vital gene that is either missing or defective in their genetic code," Fox News reported.

The rare eye disease is caused by a mutation in the CHM gene on the X chromosome and cause progressive blindness due to the deterioration of the "choroid, retinal pigment epithelium and retina." Patients with this disease usually start their lives with perfect vision and begin to experience problems with light sensitivity and peripheral vision later in life.

"It's like looking down through a telescope at a small central island of vision," MacLaren said, explain the disorder.  "And by the time they're in their 40s and 50s, they lose vision completely."

To treat the disease, researchers genetically altered an adeno-associated virus (AAV), so that it carried a corrective copy of the CHM gene. They then injected it into the retinas of six patients whom were experiencing different stages of the disease

After the treatment, the two patients who had begun to suffer vision loss showed substantial improvements in their sight.

"Now that we know the virus is doing what it should do, we need to follow the patients up and see if the improvement is correlated with the stopping of degeneration," MacLaren said.  "...We're hoping to help more people with this disease, because it's a terrible diagnosis to have."

MacLaren added that the success of the gene therapy could pave the way for the use of gene therapy on other conditions that cause vision loss. 

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