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Huntington’s breakthrough may stop disease
https://www.theguard...stating-disease
http://www.bbc.co.uk...health-42308341
Science makes life better!
The defect that causes the neurodegenerative disease Huntington's has been corrected in patients for the first time, the BBC has learned.
An experimental drug, injected into spinal fluid, safely lowered levels of toxic proteins in the brain.
The research team, at University College London, say there is now hope the deadly disease can be stopped.
Experts say it could be the biggest breakthrough in neurodegenerative diseases for 50 years.
Huntington's is caused by an error in a section of DNA called the huntingtin gene.
- Huntington's generally affects people in their prime - in their 30s and 40s
- Patients die around 10 to 20 years after symptoms start
- About 8,500 people in the UK have Huntington's and a further 25,000 will develop it when they are older
Normally this contains the instructions for making a protein, called huntingtin, which is vital for brain development.
But a genetic error corrupts the protein and turns it into a killer of brain cells.
The treatment is designed to silence the gene.
Prof John Hardy, who was awarded the Breakthrough Prize for his work on Alzheimer's, told the BBC: "I really think this is, potentially, the biggest breakthrough in neurodegenerative disease in the past 50 years.
"That sounds like hyperbole - in a year I might be embarrassed by saying that - but that's how I feel at the moment."
https://www.theguard...stating-disease
http://www.bbc.co.uk...health-42308341
Science makes life better!