Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Most HD researchers are pretty excited by the idea of 'silencing' the Huntington's disease gene, to reduce production of the harmful huntingtin protein. Two challenges - safety and delivery - are now closer to being solved thanks to collaborative work by academic and industry researchers.
Gene silencing drugs aim to slow down or prevent Huntington's disease by telling cells not to make the harmful protein. For the first time, a study has shown that gene silencing hits its target and is safe in the complex brains of rhesus monkeys.
A better understanding of the normal role of the huntingtin protein would make developing treatments easier. Surprising new results from French researchers suggest that huntingtin influences tiny hair-like structures called 'cilia'. Now we need to figure out what it means for patients.
Most Huntington's disease researchers agree silencing the huntingtin gene is one of the most promising treatments in the pipeline. But we don't know whether switching off the gene is safe. Now a Canadian team has shown that 'allele-specific' gene silencing - targeting just the mutant copy of the gene, and leaving the healthy copy active - works and is safe in an HD mouse.
The double-helix of our DNA contains a backup copy of every gene. Sometimes the backup DNA produces hidden 'messages' that can alter things in our cells. Researchers have now discovered a message in the backup copy of the Huntington's disease gene, which appears to be able to influence how much huntingtin protein is made.
What if we could edit the DNA of patients to remove the Huntington's disease mutation altogether? Sounds like science fiction, but new research in an animal model of hemophilia suggests that it can work - and now HD researchers are on the case.
Many new drugs fail because they can’t get into the brain – a major hurdle to developing new Huntington’s disease treatments. Now, researchers have used exosomes – tiny bubbles produced naturally by some cells, to 'float' gene silencing drugs across the blood-brain barrier.
Gene silencing means using specially designed molecules to 'switch off' the message that makes cells produce the harmful huntingtin protein. Our HDBuzz gene silencing primer explains the techniques, results so far and the challenges ahead.
In the first of our daily reports from CHDI’s annual HD therapies meeting in Palm Springs, we report on strategies to turn off the harmful gene and get brain cells communicating more effectively.