Natural mechanism to control Huntington's disease

Researchers have identified a natural mechanism that might help in stopping or preventing the development of Huntington's disease.

A team of scientists from Johns Hopkins University conducted experiments to block the expression of the mutated gene that causes the relentless neurodegenerative disorder.

Huntington's disease is a rare, incurable fatal disorder caused by a mutation in a single gene and marked by progressive brain damage.

Its symptoms, those typically first appear in mid-life include jerky twitch-like movements, coordination troubles, psychiatric disorders and dementia.

“The idea of being able to harness the powers of this natural mechanism for the benefit of Huntington's patients is a totally new way of thinking about therapy for the disease,” the Science Daily quoted Russell L. Margolis, a professor of psychiatry and behavioral sciences at the Johns Hopkins University School of Medicine as saying.

Currently, a major strategy among Huntington's disease researchers is to suppress the expression of the mutant gene by introducing fragments of DNA meant to bind with and sabotage the ability of the gene to make the damaging protein.

The main purpose behind this is to prevent the mutant HTT from being expressed in the brain and potentially slow the disease's growth.

The new study suggests an alternative focus involving manipulating the newly identified natural brake with a drug so that more of the brake is made that can then specifically stop or slow production of the Huntingtin protein.

The study appears online in the journal Human Molecular Genetics.