Scientists Find Molecular Switch to Prevent Huntington's Disease in Mice

[steven johnson]

There is hope for controlling the disease that devastated Woody Guthrie and destroyed half of his children

Newswise — UCLA scientists have identified a molecular switch that prevents Huntington's disease from developing in mice. Published in the Dec. 24 edition of the journal Neuron, the discovery suggests a new approach to treating the genetic disorder, which ultimately leads to death in as little as 10 years.

Affecting one out of every 10,000 Americans, Huntington's progressively deprives patients of their ability to walk, speak, think clearly and swallow. People who inherit the disorder don't show symptoms until mid-life, after many have had children and unknowingly passed on the disease. Currently, there is no effective treatment to prevent the onset or slow the progression of the disease.

Huntington's is caused by a mutation in the polyglutamine (polyQ) region of a very large protein called huntingtin. Because huntingtin is found everywhere in the body, it is a challenge to study, and the function and mechanism behind the mutant protein still remain elusive.

...that two amino acids near the beginning of the huntingtin protein can be modified by a chemical process called phosphorylation, which cells use to control protein function after the proteins have been made...The researchers found that preventing phosphorylation caused the mice to develop symptoms suggestive of Huntington's disease in humans. Mimicking phosphorylation, however, did not cause the disorder.

Scientists Find Molecular Switch to Prevent Huntington's Disease in Mice

[aquart]

1. Oh, if they could...

My breath caught just imagining.

[murielm99]

2. I hope this works out.

There is a family in my small community that has been devastated by this disease. By the time the correct diagnosis was found, it had already affected a third generation.

Knowing this family has personalized it for me. I hope they can find a real cure.

[HughMoran]

3. Imagine if they can do this in humans

Let's hope.