Could Gene Therapy Help Treat a Rare Form of Blindness?

First Posted: Jan 16, 2014 10:18 PM EST
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Gene therapy, a process that inserts copies of normal genes into a patients faulty or missing information, is a technique that has been previously used to treat rare forms of blindness, but without any success.

For the study, researchers used six patients in a trial who were suffering from choroidermia-a condition that's inherited and an untreatable form of progressive blindness. The disease occurs when a lack of protein harms the light detecting eye cells.

"We're trying to rescue cells that would otherwise have died," Dr. Robert MacLaren of the University of Oxford said, via Fox News.

Researchers injected a virus into the missing protein of the thinnest part of the patient's retinas. The study authors believe that this treatment can be very promising as there is no other way to deliver protein to the malnourished cells.

"We can do as much work as we can in the laboratory and try to sort out all the variables, but there are always surprises," said Dr. Jean Bennett of the University of Pennsylvania, according to the news organization.

However, researchers note that before the treatment goes mainstream, they will need to conduct more studies. 

"The findings are positive and do support the case for conducting more research in this area but extensive trials would be needed to establish efficacy and safety," Clara Eaglen, RNIB Eye Health Campaigns Manager notes, via The Telegraph. 

More information regarding the study can be found via the journal Lancet. 

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