Choroideremia (CHM) is a rare inherited disorder that causes progressive loss of vision due to degeneration of the choroid and retina.It occurs almost exclusively in males. In childhood, night blindness is the most common first symptom. As the disease progresses, there is loss of vision, frequently starting as an irregular ring that gradually expands both in toward central vision and out toward the extreme periphery. Researchers are looking to gene thearpy to halt the progression of this disease.- MDA
By: Maureen McFadden
It’s a condition that robs people of their sight – often starting in the teen years.
It’s a form of blindness that some call especially cruel.
People with choroideremia are born with perfect vision and then begin to lose their sight- sometimes as kids or teens.
Researchers in the United States are now building on the success of a clinical trial overseas, and are about to test a therapy designed to halt the progression of the disease, and maybe even reverse some of the damage.
In bright daylight, 10-year-old Mark Devoe has no trouble seeing his friends.
But inside, or even in the shade, Mark’s eyes sometimes don’t work.
“I have trouble seeing like trees, (or) when the road ends,” he said.
At age six, Mark’s doctors diagnosed him with the genetic condition choroideremia, which causes people to progressively lose vision until they are completely blind.
Dr. Jean Bennett, ophthalmologist and molecular geneticist at the University of Pennslvania, is one of two U.S. researchers preparing to test a gene therapy for choroideremia in humans.
“I think gene therapy holds a huge promise for developing treatments for blinding diseases,” Dr. Bennett said.
Researchers will use a virus, carrying a normal choroideremia gene and inject the virus just under the retina. The gene should begin to work in a few weeks.
“If we can prevent the disease there from continuing it would change people’s lives,” Dr. Bennett explained……….
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Source: WNDU 16