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

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?

Planting trees together: The 2016 Huntington's Disease Society of America Convention

Planting trees together: The 2016 Huntington's Disease Society of America Convention

Dr Jeff Carroll on June 13, 2016

Nearly a thousand HD family members converged on Baltimore, Maryland for the 2016 Huntington’s Disease Society of America’s Annual Convention. We normally don’t write reports from patient and family conferences, but there was something special about the atmosphere of this year’s Convention that compelled us to pen a brief update.

Huntington's Disease Therapeutics Conference 2016 - day 2

Huntington's Disease Therapeutics Conference 2016 - day 2

Dr Jeff Carroll on February 28, 2016

After an exciting day of science yesterday, day 2 saw updates on strategies to rid cells of the harmful mutant huntingtin protein and exciting reports on current and planned clinical trials.

Harnessing the power of viruses to treat Huntington's disease

Harnessing the power of viruses to treat Huntington's disease

Leora Fox on February 03, 2016

New therapies for disorders like Huntington’s disease are on the way, but getting the drugs to enter brain cells can be a major challenge. A group of scientists has redesigned and tested a harmless virus that can efficiently deliver a 'gene silencing' message throughout the brain in mice, much further than naturally occurring viruses can reach. What's more, it can be given with a simple injection into the blood, offering great potential for research in gene silencing research and beyond.

Liftoff: First humans treated with gene silencing drugs for HD!

Liftoff: First humans treated with gene silencing drugs for HD!

Dr Jeff Carroll on October 22, 2015

Today brings news that the first Huntington's Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene. These brave volunteers are the first HD patients to ever be treated with drugs designed to attack HD at its root cause, a treatment approach with huge potential. What about this news has us so excited?