13 septembre 2016 par maty185
A single injection of a new treatment has reduced the activity of the gene responsible for Huntington’s disease for several months in a trial in mice.
Huntington’s disease is a genetic disorder that affects around 1 in every 10,000 people and damages nerve cells in the brain. This causes neurological symptoms affecting movement, cognition and behaviour.
Huntington’s usually only begins to show symptoms in adulthood. There is currently no cure and no way to slow the progression of the disease. Symptoms typically progress over 10-25 years until the person eventually dies.
Now, the EU-funded FINGERS4CURE project team led by researchers at Imperial College London have engineered a therapeutic protein called a ‘zinc finger’.
Huntington’s disease is caused by a mutant form of a single gene called Huntingtin. The zinc finger protein works by targeting the mutant copies of the Huntingtin gene, repressing its ability to express and create harmful proteins.
In the new study involving mice, published in the journal Molecular Neurodegeneration, the injection of zinc finger repressed the mutant copies of the gene for at least six months.