Synthetic DNA-Based Drug Is First To Slow Progress of Huntington's Disease (theguardian.com)

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."

Comments

Re:Could it "fix" future generations?

[damn_registrars]

A single shot, or treatment. If it goes thru the bloodstream seeking copies of a gene wouldn't it find those genes in the testes/ovaries?

No. The drug - which is actually RNA based not DNA based - only interferes with messenger RNA. It essentially stops production of the Huntingtin protein (not a typo, the gene is Huntington and the protein is Huntingtin) in cells that are expressing the gene. We understand that expression of this later in life - particularly expression of the mutant form of the gene - is a direct cause of Huntington's Disease. While the function of the protein in its wild state is not fully understood, it does seem to have an important role in development. Hence we wouldn't want to shut down its expression completely in generations to come.

As for reproductive tissue, it should be unaffected as the drug is injected into the Cerebro Spinal Fluid (CSF) which usually does not reach testes and ovaries.

The treatment for future generations would probably come by way of gene editing; this is gene silencing. If you want to look up the techniques this is based on RNA interference while gene editing is more often done by CRISPR.