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

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?

New tool to measure outcomes in Huntington’s Disease clinical trials

New tool to measure outcomes in Huntington’s Disease clinical trials

Megan Krench on September 03, 2015

When patients participate in clinical trials, there needs to be some type of readout to determine whether the new treatment worked. It’s important to know two key things: What to measure and how to measure it. In the case of HD, these obstacles have vexed scientists and doctors for years. The latest research comes up with a clever new approach to overcome both challenges in a new way. These results could offer a valuable tool to study new HD therapeutics entering clinical trials.

Measuring harmful huntingtin protein in the brain’s bath water

Measuring harmful huntingtin protein in the brain’s bath water

Dr Michael Orth on May 18, 2015

Exciting technologies such as gene silencing are being developed for the treatment of Huntington’s disease. Aside from waiting for disease progression to take place, how will we know whether they are working? This has been a major hurdle for HD researchers, but we now have a super-sensitive method to measure the build-up of harmful huntingtin protein in the nervous systems of HD patients.

2015 Huntington's Disease Therapeutics Conference: Day 2

2015 Huntington's Disease Therapeutics Conference: Day 2

Dr Jeff Carroll on February 26, 2015

Our second update from the Annual Huntington's Disease Therapeutics Conference.

Huntington's disease clinical trial announcement: huntingtin-lowering drug to enter Phase I trial in 2015

Huntington's disease clinical trial announcement: huntingtin-lowering drug to enter Phase I trial in 2015

Leora Fox on November 04, 2014

A new clinical trial just announced for 2015 aims to test a “huntingtin lowering” therapy, called an antisense oligonucleotide (ASO), that attacks mutant huntingtin directly. We’re extremely excited—it’s the first-ever human HD trial to fight HD at the root of the problem, and has shown great promise in animal models. What’s the scoop?