According to the latest research, gene therapy has been found to have played a major role in adults suffering with a genetic form of childhood-onset blindness.
The study revealed such adults had some great recoveries of night vision after they were treated with gene therapy procedures.
The above result surfaced after a thorough research carried out at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.
In the original research carried out by the scientists, a group of patients suffering from Leber Congenital Amaurosis (LCA), a congenital blindness caused by mutations in the gene GUCY2D were analyzed.
The new findings are reported in the journal iScience, delivered AAV gene therapy. In the research, scientists carried out the healthy version of the gene into the retina of one eye of the affected patients.
After some days of the procedure, each patient showed signs of large increases in the eye that had gone with the procedure of gene therapy.
The above procedure is known to have mediated by some rod-type photoreceptor cells. These cells are sensitive to light and are also responsible for night blindness.
This is what lead author Samuel G. Jacobson, MD, PhD, a professor of Ophthalmology at Penn has to say, “These exciting results demonstrate that the basic molecular machinery of photo-transduction remains largely intact in some cases of LCA, and thus can be amenable to gene therapy even after decades of blindness,” he said.
Please note that LCA happens to be the most common type of congenital blindness which generally affects one in 40,000 newborn babies.
Although, the degree of vision in affected children varies, they generally show severe visual disability in their early months of illness.
Study in this regard has revealed that there are over 24 genes responsible to cause LCA.
It has been revealed that up to 20% LCA cases are caused by the disruptions due to mutations in GUCY2D. It’s a gene encoding a key protein needed in retinal photoreceptor cells for the “phototransduction cascade” — the process that converts light to neuronal signals.
The result after the above research conducted clearly indicates that GUCY2D gene therapy c=has the power to restore photoreceptor functions of rod cells found in eyes.
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