New gene therapy "could prevent blindness"

New gene therapy "could prevent blindness"

By Adrian Galbreth

A breakthrough new study could give hope to the thousands of people around the world who are struck down with a common form of blindness, through utilising gene therapy.

The treatment for X-linked retinitis pigmentosa, has been developed by experts at the University of Florida and has the potential to treat a common form of blindness that strikes both youngsters and adults.

It works through 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.

According to the report, which was published in the Proceedings of the National Academy of Sciences online, it will be some time before the therapy can be used on humans, as many complex and expensive steps remain, but the end result will have the potential to change lives.

Dr William Hauswirth, professor of ophthalmology in the UF College of Medicine and a professor and eminent scholar in department of molecular genetics and microbiology and the UF Genetics Institute, explained that providing the missing gene is one of the "ultimate ways" of treating disease and restoring significant visual function.

Around 100,000 people in the US alone have X-linked retinitis pigmentosa, which is hereditary and involves initial loss of peripheral vision and night vision, which eventually progresses to tunnel vision, then blindness.

This form of retinitis pigmentosa is the most common, and is caused by degeneration of light-sensitive cells in the eyes known as photoreceptor cells, which starts early in life, so although affected children are often born with sight, they gradually lose their vision.

Dr Alfred Lewin, a professor in the UF College of Medicine department of molecular genetics and microbiology and a member of the UF Genetics Institute, commented: "These children often go blind in the second decade of life, which is a very crucial period

"This is a compelling reason to try to develop a therapy, because this disease hinders people's ability to fully experience their world."

Further studies will now aim to advance the treatment to the human testing stage, before an eventual roll-out to the thousands of people around the world affected by the conditions.

by Alexa Kaczka

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