JERUSALEM, Feb. 27 (Xinhua) -- Israeli scientists have developed a new treatment for Huntington's disease, which can delay the onset and slow down the progression of the incurable neurodegenerative disorder, the Weizmann Institute of Science (WIS) said on Monday.
Huntington's disease is a rare hereditary disorder that causes brain cells to die and leads to motor, mental, and psychiatric problems.
The treatment, described in the journal EMBO Molecular Medicine, involves two small molecules that can cross the blood-brain barrier, a protective layer that blocks most drugs from reaching the brain, and target the faulty gene that produces the harmful huntingtin protein, which is believed responsible for Huntington's disease.
The researchers tested the molecules on human cells and genetically modified mice with a human version of the mutant gene. They found that the molecules reduced the abnormal protein levels without affecting the normal one, and slowed down the disease's progression, reversing some symptoms and reducing brain damage.
The team also showed that the molecules could be delivered orally or by injection, avoiding the risk of direct brain administration. They said the treatment delayed the onset of the disease even when given at an early stage. ■
