Scientists cure brain wasting disease using gene therapy

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Sydney, Mar 15 (UNI) Scientists are a step closer to curing brain-wasting disease using gene therapy.

The treatment involves injecting a modified form of the HIV virus into the affected region of the brain. The virus infects cells and delivers a gene that can fix the genetic fault causing the symptoms.

So far, the feat has only been achieved in mice, but Japanese researchers said the animals' symptoms of uncoordinated movement "markedly improved". It raises the possibility of, a gene therapy for humans.

The scientists are targeting a rare inherited neurodegenerative condition, spinocerebellar ataxia. It can manifest in various forms leading to uncoordinated movement and speech as well as problems with vision and swallowing. Symptoms typically begin in late childhood or early adult life and they become progressively worse, leading to death. There is no cure.

"It's like being drunk without having the pleasant effects and it's extremely disabling and slowly gets worse," said professor Patrick Chinnery, an expert in neurogenetics at Newcastle University as saying in The Guardian. "These people eventually aren't able to feed themselves, wash themselves and they end up in a wheelchair.

All we can offer them in the clinic is a diagnosis, advice about the chances of it happening again in the family and supportive care." The disease is one of a family of inherited neurodegenerative conditions including Huntington's which could all be susceptible to the same approach. These diseases affect around one in 5,000 of the UK population. Each disease is caused by mutations to different genes, but the type of genetic stutter that causes them is the same.


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