Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.

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Articles with the topic: gene-silencing

HDSA’s Q&A on the latest huntingtin-lowering update from Ionis and Roche

HDSA’s Q&A on the latest huntingtin-lowering update from Ionis and Roche

Leora Fox on April 26, 2018

Earlier this week, Ionis Pharmaceuticals put out a press release after presenting data from the HTTRx (RG6042) Huntingtin-lowering clinical trial program at a scientific conference. Ionis’ partner Roche also released a community statement. Our friend at Huntington’s Disease Society of America, Dr Leora Fox, has prepared this Q&A in response to community questions received after the release. We liked it so much, we decided to reproduce it here, by kind permission, for the benefit of HDBuzz readers.

Huntington's disease therapeutics conference 2018 - day 3

Huntington's disease therapeutics conference 2018 - day 3

Dr Jeff Carroll on March 02, 2018

Good morning from the final day of the 2018 HD Therapeutics Conference! Two sessions today, the first focused on the protein made from the HD gene. The second includes updates on Huntingtin Lowering Trials from both Wave Life Sciences and Ionis Pharmaceuticals.

An early role for the Huntington's disease gene – but don't believe all the headlines

An early role for the Huntington's disease gene – but don't believe all the headlines

Dr Jeff Carroll on February 27, 2018

New hints are emerging about the normal role of the gene that causes Huntington's disease. A recent report uses cutting edge techniques to study this question in cells growing in the lab. We'll help separate the fascinating new science from some scary-sounding headlines.

Success! ASO drug reduces levels of mutant protein in Huntington's disease patients

Success! ASO drug reduces levels of mutant protein in Huntington's disease patients

Dr Jeff Carroll on December 11, 2017

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.

Update confirms Huntington's disease 'gene silencing' trial on track

Update confirms Huntington's disease 'gene silencing' trial on track

Dr Jeff Carroll on October 15, 2017

Ionis Pharmaceuticals launched the first ever trial of a huntingtin-lowering drug – sometimes called a 'gene silencing drug' – in late 2015. In a significant update, the company has announced two important milestones: the trial is now fully recruited, and an 'open-label extension' will be activated for the volunteers in the current trial. While nothing is guaranteed, this bodes well for the future of this important program.

New roles for huntingtin: removing a healthy protein to understand its function

New roles for huntingtin: removing a healthy protein to understand its function

Leora Fox on September 26, 2017

Some techniques aimed at lowering mutant huntingtin can also affect the normal form of the protein. With clinical trials underway, it’s all the more important to understand the role of normal huntingtin in the adult brain. Researchers recently inactivated the huntingtin gene in healthy adult mice of different ages. They found that this could cause neurological and behavioral problems. Mice aren’t perfect for modelling human brains, and no huntingtin-lowering drug would remove the protein completely - but this research supports the need for continued caution as we test drugs that lower normal huntingtin.

Precision huntingtin-lowering drug trials target the mutant protein

Precision huntingtin-lowering drug trials target the mutant protein

Dr Michael Flower on August 25, 2017

A new exciting chapter in Huntington’s disease (HD) treatment is just beginning – WAVE Life Sciences have announced PRECISION-HD1 and 2, clinical trials of two new drugs that lower the mutant Huntington’s disease protein. We’re excited about this novel approach to huntingtin lowering therapies, but these are early days and we’ve got a long way to go to show they’re safe and effective in people.

Huntington's Disease Therapeutics Conference 2017 - Day 2

Huntington's Disease Therapeutics Conference 2017 - Day 2

Dr Jeff Carroll on April 29, 2017

Day two of the conference looks at some of the most promising approaches to fighting Huntington's disease.

Important advances in next generation genome editing tools for Huntington's Disease

Important advances in next generation genome editing tools for Huntington's Disease

Mr. Shawn Minnig on October 31, 2016

Recent days have seen a slew of news emerging regarding the use of something called genome editing as a potential therapy for genetic diseases like Huntington's Disease. These approaches, which include exotic sounding tools like zinc finger nucleases and CRISPR/Cas9, differ from more traditional ways reducing the impact of the HD mutation on cells. What's new in this exciting area of research?