University hope over childhood dementia
UNIVERSITY of Manchester scientists have developed a breakthrough gene therapy they hope will treat a rare brain disease dubbed childhood Alzheimer’s.
Sanfilippo syndrome is a devastating genetic disorder which affects about one in 85,000 people.
It can affect children as young as three, destroying brain cells and leaving sufferers unable to walk, talk or swallow. Most people with the condition die in their late 20s.
There’s no known cure, but now there’s hope that a specially modified virus developed in Manchester could help treat the ‘type C’ form of the ‘horrible’ disease.
The virus has been specifically altered to deliver the missing gene to the brain. Trials conducted with mice have shown ‘complete behavioural and brain correction.’
Scientists now plan to carry out clinical trials in the near future.