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

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Switch off the genome editor when you’re done

Switch off the genome editor when you’re done

Dr Michael Flower on November 02, 2017

Genome editing is a much-discussed frontier in medical science at the moment, with ‘DNA surgery’ having the potential to treat or cure genetic diseases like Huntington’s. Here we look at what this technology can currently do and discuss the challenges that still lie in the way. We’ll also discuss how a team of Swiss scientists have recently developed a way to switch off the genome-editing machinery after it’s done its job.

Latest news

A new way of thinking about trials to prevent Huntington's disease

A new way of thinking about trials to prevent Huntington's disease

Dr Jeff Carroll on October 27, 2017

A new analysis of clinical data from the TRACK-HD and COHORT studies proposes a way to design of clinical trials designed to delay the onset of HD, rather than treating symptoms after they occur.

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.

A step forward for gene editing: CRISPR-Cas9 and HD

A step forward for gene editing: CRISPR-Cas9 and HD

Leora Fox on July 26, 2017

CRISPR-Cas9 is an experimental gene editing technique used to make precise changes in DNA. For the first time, scientists have used this approach to attack the Huntington's Disease mutation in the brain cells of a mouse. Other researchers are refining CRISPR-Cas9 to be more efficient, specific, and safe. It’s still a long way from use in HD patients, but its application in mice is an exciting step forward.

New study reveals a potential HD biomarker

New study reveals a potential HD biomarker

Leora Fox on June 07, 2017

What if a blood test could provide information about the status and course of HD? This is the premise of seeking HD biomarkers; they may someday help guide treatment decisions and predict how symptoms will change. A team of researchers spanning multiple countries recently analyzed blood, brain images, and clinical exams from the TRACK-HD study. They found that blood levels of a protein called neurofilament light chain corresponded with the severity of HD, making it a potential biomarker.

Has a "wonder drug" for dementia been discovered? (Spoiler alert: no.)

Has a "wonder drug" for dementia been discovered? (Spoiler alert: no.)

Dr Michael Flower on May 07, 2017

If media reports of a "wonder-drug" that could "stop all neurodegenerative brain diseases, including dementia” seem too good to be true, that's because they are. The truth behind the headlines is that researchers tested thousands of already-licensed drugs in worms, and a couple that went on to show beneficial effects in mouse models of two rare forms of dementia. While it gives researchers two new leads, this research doesn't prove anything about these drugs in patients with neurodegenerative diseases, and has virtually nothing to do with Huntington's disease at all.

Huntington's Disease Therapeutics Conference 2017 - Day 3

Huntington's Disease Therapeutics Conference 2017 - Day 3

Dr Ed Wild on April 30, 2017

The final day of the 2017 Huntington's Disease Therapeutics conference brought updates on research to better understand how HD affects brains, and breakthroughs in using brain cells to understand and treat HD.

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.