Sentences with phrase «reducing mutant huntingtin»

Next steps will be to conduct efficacy studies to show whether reducing mutant huntingtin can slow down disease progression.

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.

Not exact matches

University of California, Irvine neurobiologists Leslie Thompson and Joseph Ochaba with the Departments of Neurobiology & Behavior and Psychiatry & Human Behavior and their colleagues from UCI and from Children's Hospital of Philadelphia have shown that reducing the aberrant accumulation of a particular form of the mutant Huntingtin protein corresponds to improvement in symptoms and neuroinflammation in HD mice.

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 phase 1 / 2a study of IONIS - HTTRx was recently completed and showed the therapy to be safe and well - tolerated but also showed it's ability to reduce the level of mutant huntingtin in a dose dependent manner in Huntington's Disease patients.

Activity of the mutant huntingtin gene was reduced by around 50 % in the brain near the injection site.

The appearance of blobs of mutant huntingtin protein was reduced by about 40 %, too.

We hope this will allow WAVE to actually measure what they're trying to do, which is to reduce the levels of the mutant huntingtin protein in the brain.

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