Researchers move one step closer to Huntington's gene silencing

Researchers have moved closer towards silencing the Huntington's disease gene by solving the challenges of safety and delivery.

Gene silencing uses a specifically designed drug to intercept RNA, which is a message molecule produced from the Huntington's disease gene that tells cells to make the harmful protein that causes Huntington's.

Currently the treatment has been successfully tested in several animal models, indicating that the brain can actually recover from some of the symptoms of Parkinson's by lowering protein levels.

Nonetheless, before human trials could occur researchers had to solve the problems of safety and delivery, as there may be unexpected side effects and introducing the drug directly into the nervous system can be difficult.

However, three studies have now used RNA interference (RNAI) drugs in primates successfully and safely to block RNA.

RNAi introduces homologous double stranded RNA to target a gene's product and nullify effects.

What's more, a study in Kentucky used convection enhanced delivery to inject the drug, by placing tubes through the skull into the brain.

Find out how Barchester works to create safe and understanding environments for those living with Huntington's disease.