Sentences with phrase «lowering mutant huntingtin»

There are many ways of achieving the goal of lowering mutant huntingtin, expected to start new trials soon or already in early trials.

First things first - the news that IONIS - HTTRx lowers the mutant huntingtin protein is great - but it is not a cure.

Not exact matches

Being able to detect and measure the amount of mutant huntingtin present in the nervous system will be a valuable way of seeing whether the gene - silencing drug is hitting its target and has the intended effect, lowering the amount of disease causing mHTT protein.

2015 will see the start of the first human clinical trial of a gene silencing or huntingtin - lowering drug, which specifically aims to reduce production of mutant huntingtin in the brains of HD patients.

The test, called a «single molecule counting assay», combines fluorescent antibodies with a laser detection chamber to count individual molecules of mutant huntingtin with a very low detection threshold.

In an announcement likely to stand as one of the biggest breakthroughs in Huntington's disease since the discovery of the HD gene in 1993, Ionis and Roche today announced that the first human trial of a huntingtin - lowering drug, IONIS - HTTRx, demonstrates that it reduces mutant huntingtin in the nervous system, and is safe and well - tolerated.

Some of these approaches to attack mutant huntingtin also reduce levels of the normal protein, including the Ionis huntingtin - lowering drug.

That's not what we expect when patients are given huntingtin - lowering drugs, which might produce around 50 - 75 % reductions in the mutant and healthy protein.

Doing that lowered levels of the protein for which the gene is a recipe: mutant huntingtin.

We know that lowering the level of mutant huntingtin protein in HD mouse models significantly improves symptoms reminiscent of HD, providing hope that similar treatments in people may be effective.