Researchers may finally have tracked down an explanation for expansions of three-letter DNA sequences that disrupt genes, and lead to Huntington’s disease and numerous other neuromuscular and neurodegenerative conditions. The study, “The role of break-induced replication in large-scale expansions of (CAG)n/(CTG)n repeats,” published in the journal Nature Structural & Molecular…
News
Huntington’s disease patients’ inability to recognize people’s emotions from their facial expressions appears to be linked to dysfunction in more than one area of the brain, according to a study. The brain areas include “emotion–related regions, such as front-striatal networks and limbic areas, and regions associated with visual processing,” according…
Researchers have developed a new way to deliver therapies to areas of the brain they have been unable to reach due to the blood-brain barrier that protects the brain from microorganisms. The technique may lead to new treatments of neurological disorders, including Huntington’s disease, according to the research. The study,…
Researchers have identified a molecular mechanism in stem cells that may delay the onset of aging-related diseases associated with the production of abnormal proteins, according to a study published in the journal Nature Communications. This finding may help design new therapeutic tools to treat diseases characterized by poorly regulated…
A study using a mouse model suggests that some symptoms of Huntington’s disease are caused by muscle abnormalities, and are not related to dying neurons. The findings may present new opportunities for improving patient care by using muscle-specific drugs. The study, “Progressive Cl− channel defects reveal disrupted…
Researchers have identified a new mechanism of cell death in Huntington’s disease called “ballooning cell death” (BCD), according to new research from Tokyo Medical and Dental University. The study, “Targeting TEAD/YAP-Transcription-Dependent Necrosis, TRIAD, Ameliorates Huntington’s Disease Pathology,” was published in the journal Human Molecular Genetics. Huntington’s…
Huntington’s disease is caused by excessive repeats of a portion of DNA, called CAG triplets, within the gene that codes for huntingtin (HTT). New research shows how the number of CAG repeats determines the final form and the aggregation profile of the resulting protein. The study, “The Aggregation Free Energy…
Gene therapy biotech uniQure is prioritizing its drug pipeline to develop treatments for Huntington’s disease, hemophilia B, and its partnered gene therapy programs associated with uniQure’s collaboration with Bristol-Myers Squibb (BMS) in cardiovascular disease. The company will restructure its research and development organization in the Netherlands and consolidate manufacturing in…
Researchers identified a new molecular mechanism related to mutant huntingtin (HTT) — messenger RNA — that may play a role in the development of Huntington’s disease. This finding may help define new therapeutic approaches to tackle the disease. The study, “Targeting CAG Repeat RNAs Reduces Huntington’s Disease Phenotype Independently Of…
Researchers identified several new biomarkers in mice that may help in the early diagnosis of Huntington’s disease in humans, according to a new study. These biomarkers may also be useful to evaluate patients’ responses to potential treatments. The Stanford University School of Medicine study, “Potential Biomarkers To Follow The…
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