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

Are you looking for our logo? You can download our logo and get information about how to use it on our sharing page

Articles with the topic: mouse-model

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.

Enemy at the gates – huntingtin disrupts nuclear transport

Enemy at the gates – huntingtin disrupts nuclear transport

Tom Peskett on April 21, 2017

Exciting new studies provide evidence that a particular kind of cellular trafficking goes awry in Huntington's Disease. Specifically, researchers have learned that traffic in and out of the cells control center - the nucleus - breaks down in HD. These findings open up new avenues for HD research.

The benefits of migration, highlighted in Huntington's disease

The benefits of migration, highlighted in Huntington's disease

Dr Michael Flower on February 10, 2017

Huntingtin, the protein responsible for Huntington’s disease, is fundamentally important for fetuses to develop in the womb, but we don’t know yet exactly what part it plays in this intricate process. Normally, neurons start life deep within the developing brain, migrate out to the surface and then make a network of connections with others, but Sandrine Humbert’s group showed that those without huntingtin get stuck, never making it to where they need to go. Neurons with mutated huntingtin are no better than those that lack it completely. However, reintroducing normal huntingtin, or the proteins through which it acts, allows neurons to migrate normally again, offering tantalising new ways to treat Huntington’s disease.

Early exposure to the HD protein may cause life-long symptoms

Early exposure to the HD protein may cause life-long symptoms

Mr. Shawn Minnig on June 28, 2016

When the ‘healthy’ HD gene functions as it should, one of its many jobs is in the development of normal embryos. Researchers have long assumed that the ‘mutant’ HD gene inherited by people with HD is still able to do this job, since HD patients develop normally and don’t show signs until later in life. A surprising new finding suggests we may have to think carefully about this assumption!

Huntingtin takes a trip: harmful proteins pass between brain cells

Huntingtin takes a trip: harmful proteins pass between brain cells

Leora Fox on June 06, 2016

Clumps of mutant huntingtin protein in brain cells are a hallmark of HD, and they build up slowly, occupying more and more cells over time. Recent research in mice shows that the harmful proteins can travel between neurons, setting off a chain reaction that leads to more sick cells and the development of symptoms.

A recycled drug gives us new insight in to HD

A recycled drug gives us new insight in to HD

Leora Fox on December 15, 2015

Researchers have found a connection between HD and an energy-regulating protein called PPAR-delta. Giving PPAR-delta a boost with an existing drug was protective in HD cells and mice, but we’ll likely need to research and test it further before it can go to the HD clinic.

A multiple sclerosis drug that works for Huntington's disease: the real deal or too good to be true?

A multiple sclerosis drug that works for Huntington's disease: the real deal or too good to be true?

Melissa Christianson on November 24, 2015

Thinking problems in Huntington’s disease take a huge toll from early in the disease. Now, new work suggests that a drug already approved by the FDA to treat another brain disease – multiple sclerosis – may stave off these problems in HD mice. Could these results be real, or are they too good to be true?

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.

A faulty filter? Blood vessel changes in Huntington's disease

A faulty filter? Blood vessel changes in Huntington's disease

Melissa Christianson on May 13, 2015

Because brain cell death drives symptoms in Huntington’s disease, these cells steal the spotlight in disease-related research. But new research on the brain's blood supply has uncovered changes in HD that could be making it harder for the brain cells to cope with the disease.