Huntington's disease affects approximately 1/10,000 of the general population. The genetic cause is a flaw in chromosome 4. In the normal case there are 4-35 repeats of the CAG sequence.

The number of CAG repeats directly affects the severity of the disease. Individuals with a higher number of extra repeats usually experience earlier onset of symptoms.

Successes in some Animal studies involving fetal tissue transplant into the striatum suggested a possibility that transplant treatments might be effective in humans. Several early attempts included some positive reports, however there had been no proper or rigorous protocols developed or completed. Studies with complete and agreed protocols are now in progress. Research has also been performed in porcine tissue transplants.

Huntington's researchers are also engaging in drug discovery methods, looking at both gene therapy methods and means to either inhibit the malfunction of the damaged huntingtin protein or help the body to fight the problem.