John.Banister writes: The Guardian reports of early success in the trial of a synthetic DNA based drug, Ionis-HTTRx, at University College London’s Huntington’s Disease Center. Bionews explains that this gene silencing drug binds to the RNA transcript of the faulty huntingtin gene, triggering its destruction before it can go on to make the huntingtin protein. There’s much excited speculation that the same technique could be used for Alzheimer’s and Parkinson’s disease, once people know which genes to target. “The trial involved 46 men and women with early stage Huntington’s disease in the UK, Germany and Canada,” reports The Guardian. “The patients were given four spinal injections one month apart and the drug dose was increased at each session; roughly a quarter of participants had a placebo injection. After being given the drug, the concentration of harmful protein in the spinal cord fluid dropped significantly and in proportion with the strength of the dose. This kind of closely matched relationship normally indicates a drug is having a powerful effect.”
Read more of this story at Slashdot.
Source: Slashdot – Synthetic DNA-Based Drug Is First To Slow Progress of Huntington’s Disease
