Initial results revealed that a genetic treatment was injected into patients with the slowdown of Huntington’s disease by 75%. Huntington’s disease is a degenerative nervous condition, in which neurons decompose in certain areas of the brain.
The results of this small initial experiment, which conducted human beings, are the first results of a treatment attempt that shows a concrete benefit in patients. The data was not subject to the review of the peers or published in an academic magazine, and therefore it should be considered an initial indication of what the final results may show.
UNIQURE – the company -based searcher in Amsterdam in the Netherlands – announced the results on September 24, and wrote about the New York Times.
Huntington’s disease is a rare brain disease characterized by motor, cognitive and emotional symptoms, and ultimately leads to premature death. The disease occurs due to a mutation in one gene, called Huntington.
Each person’s brain cells produce Huntington protein, but for people with the disease this genetic mutation stimulates nerve cells to produce a toxic version.
These abnormal proteins accumulate, and over time damage the brain cells and destroy them.
Silence the disease
Symptoms of Huntington disease usually appear in middle age, and include involuntary movements, especially in the ends, face and tongue. With symptoms, it can cause difficulty walking, talking and swallowing.
Cognitive and emotional symptoms, including depression, emotion, problems in planning, attention and self -control, are also common. Patients may die within 10 to 30 years of symptoms.
The genetic therapy directed the neurons to produce a riotyan (RNA) (RNA) (RNA) associated with Huntington’s rural nuclear acid, which prevents cells from producing defective protein.
Treatment was provided by a genetic average virus that is surgically injected into the brain. The virus carries a special piece of DNA inaugurated DNA. The neurons are directed to produce the roller acid.
“What we do is to reprogram neurons to become small factories that produce their own medicine,” said Dr. Ed Wilde, a professor of neuroscience at the University of London.