Huntington's disease is caused by carrying a faulty version of the gene encoding the Huntingtin protein. Multiple extra copies of a short repeated sequence in the gene lead to an expanded mid-section of the protein, which causes major problems in nerve cells.
Now scientists at the Centre for Genomic Regulation in Spain have shown that it's not just the faulty expanded protein that causes disease, as was previously thought, but also the RNA messenger itself that is read from the gene when it is switched on.
Writing about their findings in The Journal of Clinical Investigation, the team showed that interfering with the expanded section of the gene at the level of the messenger RNA could make a difference to the signs of disease in mice with a model of the illness.
Huntington's disease is currently incurable, so the team hopes their findings will open the door to new approaches for treatment based on targeting the Huntingtin RNA as well as the faulty protein.