"The first drug that can potentially correct the underlying defect that causes Huntington's disease has been taken by patients in a clinical trial. The disease is caused by the brain producing a mutant protein called huntingtin which damages and ultimately kills off brain cells. As Huntington's progresses it leads to uncontrolled movements, behavior changes and poor cognition. Life expectancy after diagnosis can be as short as 10 years."
The drug used in the clinical trial is known as a gene silencer. It works by targeting mRNA (messenger RNA) strands which contain the instructions for the mutated protein. The drug was manufactured to bind to the mRNA and neutralize it. The drug will be administered via injections into the spinal cord over the course of four months and then the patients will be observed for an additional three months.
This is an amazing step forward in the field of medicine. Not only is Huntington's incurable to date, but the medications for it only treat the symptoms while doing nothing to slow the damage. The article goes on to discuss the safety precautions the clinicians are taking while administering the drug. Hopefully, the results for the human trials are as promising as the pre-clinical work.