Experimental Drug For Huntington's Disease Jams Malfunctioning Gene
More than 600 people will take part in study to test a promising treatment for Huntington's disease, a fatal inherited condition. The experimental drug interferes with defective genetic machinery.
Scientists are gearing up a major study to find out whether a drug can silence the gene that causes a devastating illness called Huntington's disease.
This development follows the discovery that the experimental drug reduced levels of the damaged protein that causes this mind-robbing ailment. The new study will determine whether that drug can also stop progression of the disease.
It is also another sign that drugs built with DNA, or its cellular collaborator RNA, can be powerful tools for tempering diseases that until now have seemed out of reach.
Huntington's disease is an apt target because it's caused by a single mutated gene. It also a frightening and devastating disease.
The symptoms
