New Treatment Halts Huntington's Disease Activity in Mice

Article: http://www.sciencealert.com/activity-of-the-huntington-s-disease-gene-has-been-halted-for-6-months-in-mice

Research Study: http://molecularneurodegeneration.biomedcentral.com/articles/10.1186/s13024-016-0128-x


Researchers have successfully tested a new treatment option that stops the activity of Huntington's for an extended period of time. Huntington's is the result of a mutation in the Huntingtin gene. It is caused by an abnormally large number of CAG repeats, which produce a mutant form of the protein. It affects brain function and causes twitching/jerking.

The treatment includes an injection of a Zinc Finger protein that binds to a DNA sequence. In this study, the used a ZF protein to bind to the CAG tri-nucleotide repeats on the Huntingtin gene in the fourth chromosome. The protein's purpose is to represses the expression of the gene, which in theory should lower the amount of potentially harmful proteins that cause Huntington's.

In mice, the treatment repressed more than 50% of the mutant gene for 6 weeks, and still showed repression after 6 months (23%).

There is no cure for Huntington's and the exact cause in unknown, but researchers think that by attacking the expression of the gene itself, they can slow the progression of the disease. In order to do that, they must try to see how repressing the gene is affecting the symptoms of Huntington's and if the symptoms themselves are being halted long-term or not. They have strong evidence for that, so we may see human clinical trials in as soon as 5 years!




Arash Panjwani