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Researchers Develop Gene Therapy That Could Correct a Common Form of Blindness

A new gene therapy method developed by University of Florida researchers has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function.

Several complex and costly steps remain before the gene therapy technique can be used in humans, but once at that stage, it has great potential to change lives.

The researchers tackled a condition called X-linked retinitis pigmentosa, a genetic defect that is passed from mothers to sons. Girls carry the trait, but do not have the kind of vision loss seen among boys. About 100,000 people in the U.S. have a form of retinitis pigmentosa, which is characterized by initial loss of peripheral vision and night vision, which eventually progresses to tunnel vision, then blindness. In some cases, loss of sight coincides with the appearance of dark-colored areas on the usually orange-colored retina.

The UF researchers previously had success pioneering the use of gene therapy in clinical trials to reverse a form of blindness known as Leber's congenital amaurosis. About 5 percent of people who have retinitis pigmentosa have this form, which affects the eye's inner lining.

The X-linked form of retinitis pigmentosa addressed in the new study is the most common, and is caused by degeneration of light-sensitive cells in the eyes known as photoreceptor cells. It starts early in life, so though affected children are often born seeing, they gradually lose their vision.

The UF researchers and colleagues at the University of Pennsylvania performed the technically challenging task of cloning a working copy of the affected gene into a virus that served as a delivery vehicle to transport it to the appropriate part of the eye. They also cloned a genetic "switch" that would turn on the gene once it was in place, so it could start producing a protein needed for the damaged eye cells to function.

After laboratory tests proved successful, the researchers expanded their NIH-funded studies and were able to cure animals in which X-linked retinitis pigmentosa occurs naturally. The injected genes made their way only to the spot where they were needed, and not to any other places in the body. The study gave a good approximation of how the gene therapy might work in humans.

The researchers plan to repeat their studies on a larger scale over a longer term, and make a version of the virus that proves to be safe in humans. Once that is achieved, a pharmaceutical grade of the virus would have to be produced and tested before moving into clinical trials in humans. The researchers will be able to use much of the technology they have already developed and used successfully to restore vision.

Sources: http://www.sciencedaily.com/releases/2012/01/120123163412.htm

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Reflections:

Eyesight loss is rather common in this world. Having inventing this therapy, it will definitely put a dent in people's lives. Although this therapy has not been published, I'm sure that it will work wonders. Now, mankind is taking one step towards finding a cure for the blind. Imagine yourself leaving in a world of darkness. Imagine that you have found this cure. Wouldn't you be excited?

However, as said by the article, this therapy is very costly and poorer families might not be able to afford it. Therefore, this new discovery might not cater to the majority as only the wealthy may be able to afford this kind of treatment. Maybe a possible way to counter this would be to get hospitals to raise funds for the poor and offer this treatment to them at a subsidised price.

After this article, I also realised that technology is advancing rapidly and that medical Science is also improving. With more amazing cures being made, I am sure that scientist may one day be able to cure the blind completely. I feel that these scientists should be recognised since they are working so hard to find new inventions for Earth. This new cure is also reliable since it has been tested on animals. However, I still feel unsure of this research since it has not been proven to be effective against humans. If I were the scientist, I would get a live human specimen to demonstrate to the world the effectiveness of this cure. In conclusion, I feel that this therapy will bring joy to many.