Huntington’s disease is a neurological disease that combines dementia and motor neurone issues, making for a very unpleasant experience. Historically, people suffering from Huntington’s disease had low prospects for living their lives as they normally did and a shorter lifespan. A UK team has developed a very interesting solution to treat Huntington’s disease by injecting modified viruses directly into the brain, the really cool thing is that these little viruses then block the ability of Huntington’s disease to spread. It’s a novel and very 21st century approach and good news not only for people who may get Huntington’s but also for related neurological diseases.
It starts with a safe virus that has been altered to contain a specially designed sequence of DNA.
This is infused deep into the brain using real-time MRI scanning to guide a microcatheter to two brain regions – the caudate nucleus and the putamen. This takes 12 to 18 hours of neurosurgery.
The virus then acts like a microscopic postman – delivering the new piece of DNA inside brain cells, where it becomes active.
This turns the neurons into a factory for making the therapy to avert their own death.
The cells produce a small fragment of genetic material (called microRNA) that is designed to intercept and disable the instructions (called messenger RNA) being sent from the cells’ DNA for building mutant huntingtin.
This results in lower levels of mutant huntingtin in the brain.