Inherited blindness treatable via gene therapy
genetic blindness

People born with a form of genetic blindness may finally experience vision after researchers discovered that retinal gene therapy can cause their eyesight to increase by 1,000 times.

In a study involving three adults between the age groups of 21-42 it was observed that one year after being injected with corrective genes into their retinas, the vision developed by 1,000 times. One of these patients could even read a digital clock for the first time.

Leber congenital amaurosis type2(LCA2), is a rare form of blindness in which the photo-receptor cells can not respond to any sort of light due to the lack of production of essential proteins by a gene known as RPE65.

These patients were born with the LCA2 type of blindness and to initiate the production of the essential proteins in their eyes, scientists used a harmless virus found in most people which enabled the gene-RPE65 to reach a part of their retina. This gene finds its way into the cells that were missing it where it begins creating the protein which is actually an enzyme that consequently restores the vision.

People with vision have a central region which is instrumental in normal vision but in these patients treated with the gene therapy an altogether new region is created which becomes more sensitive to light.

The brains of these patients treated with gene therapy, noticed this new location in the retina and rearranged the eye muscles to a state that they could use either the central region of the retina or this newly created part of the retina depending on the brightness of light.

Dr. William Hauswirth, professor of ophthalmology at the University of Florida College of Medicine said, "What's truly astounding is the brain, even in an adult, is still adaptable enough to learn to use these regions of the retina,"

The study was led by Dr. Samuel G. Jacobson, professor of ophthalmology at the University of Pennsylvania and was supported by the National Eye Institute.

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