Articles tagged with Huntingtons Disease
This technology enables high-throughput screening for small molecules that selectively lower toxic CAG repeat RNA, helping to develop therapies for neurodegenerative diseases like Huntington’s and spinocerebellar ataxias. The system overcomes major limitations of previous constitutive systems by utilizing an inducible promoter, dual-re...
A new atlas of the brain’s striatum has identified 31 subgroups of neurons, including those involved in addiction, depression, and schizophrenia. The atlas may help scientists develop new drugs to combat these conditions, particularly by targeting specific cell populations affected by Huntington’s disease.
Researchers suggest that targeting tiny protein aggregates known as oligomers, which form early in the disease process, may lead to breakthroughs in treating protein-misfolding diseases. Oligomers are increasingly believed to drive cellular damage, and their early formation may be more toxic than large protein deposits.
Researchers developed a novel imaging approach called SANDI to study microstructural brain abnormalities associated with Huntington's disease. The model revealed differences in cell density and size, correlating with disease progression and motor performance.
Researchers at the University of Illinois developed a gene editing tool to treat Huntington's disease by altering a specific point in the huntingtin gene. The treatment reduced toxic protein fragments, symptoms, and brain degeneration in mice, providing a new approach for treating genetic diseases.
A landmark study identifies new genetic risk factors associated with fibromyalgia syndrome, pointing to a neurological origin of the disorder. The research found substantial overlap between fibromyalgia and other conditions, suggesting shared biological mechanisms that may benefit from targeted therapies.
The REGEN4HD study marks a significant milestone in the development of regenerative medicine approaches for Huntington's disease. The Phase 1b/2a trial enrolls early-stage symptomatic patients to evaluate safety and tolerability.
A new study describes a gene therapy strategy that uses the brain's glymphatic transport system to distribute engineered viral vectors throughout the brain. This approach enables preferential targeting of human glial cells while minimizing exposure to other cell types and organs, addressing two major challenges in neurological medicine.
A study identified a brain pattern related to the tau protein that changes according to the stage of Huntington’s disease. The discovery opens doors to new biomarkers and treatments for this rare, hereditary neurodegenerative disease.
Researchers identified disrupted neural circuits behind Huntington's disease and used optogenetics to selectively activate key neurons, restoring function and improving motor skills. The study provides hope for future therapies targeting specific brain circuit imbalances.
A new study published in EMBO Molecular Medicine shows that anle138b can significantly reduce toxic protein clumps in the brain and alleviate symptoms of Huntington's disease. The compound also addresses the underlying cause of the disease by preventing disease-specific harmful protein aggregates.
Researchers from Florida Atlantic University have identified a key immune pathway that appears to drive damaging inflammation in Huntington disease. Blocking this pathway reduced brain inflammation, protected neurons, and improved movement in a humanized mouse model of the disease.
A recent study by Dr. Walaa Oweis and Prof. Eran Meshorer found that protein clumps in the brain may actually be protecting neurons from stress, acting as a vital quarantine system.
Huntington's disease is caused by a toxic protein that builds in brain cells and spreads to other cells through tunneling nanotubes. Disrupting this pathway reduces the spread of the disease-causing protein, suggesting a new target for therapy.
A study reviews decades of research on Hsp70's role in neurodegenerative diseases, highlighting its protective effects and potential therapeutic value. The review reveals that different Hsp70 isoforms interact with co-chaperones and cellular pathways to determine protein clearance.
Researchers have identified a key protein that produces hydrogen sulfide gas as a therapeutic target for Alzheimer's disease. Experiments in genetically engineered mice show that this protein, Cystathionine γ-lyase (CSE), plays a critical role in cognitive function and memory formation.
Researchers developed a computational tool to identify genetic vulnerabilities in memory-making brain cells linked to Alzheimer's. The 'seismic' algorithm integrates genetic data with single-cell RNA sequencing, revealing a detailed picture of affected cell types and their genetic programs.
Researchers found that increased levels of EPS8 drive pathological protein aggregation and neurodegeneration in worms and human cell models. By reducing EPS8 activity, they prevented toxic protein accumulation and preserved neuronal function.
Dr. David Rubinsztein shares his personal journey from childhood curiosity to discovering autophagy, a natural process that clears toxic proteins causing devastating neurodegenerative diseases. His research has established autophagy upregulation as a viable therapeutic strategy for conditions affecting millions worldwide.
Researchers developed a digital motor score, HDDMS, using smartphone apps to track disease progression in Huntington's disease. The HDDMS is around twice as sensitive as the current clinical measure, allowing for more efficient clinical trials and potentially accelerating drug development.
Dr. Vanessa Casha, a postdoctoral scholar at the UCLA Brain Research Institute, received the 2025 Hereditary Disease Foundation's Nancy S. Wexler Young Investigator Prize for her research on Huntington's disease. She aims to identify key proteins driving the disease and potential therapeutic targets.
Researchers at the Broad Institute developed a gene editing approach that interrupts and stabilizes trinucleotide repeat expansions, which cause Huntington's disease and Friedreich's ataxia. The method, using base editing, prevents the repeats from growing in length, halting or slowing down disease progression.
Davis Joseph's groundbreaking discovery identifies a common master switch that can cure multiple brain-related diseases with a single method. The unified theory establishes that regulating axon-based 4E-BP2 protein deamidation can control disease progression.
Researchers have discovered that the adult brain can generate new neurons that integrate into key motor circuits, potentially reversing damage in Huntington's disease. These newly generated cells replace lost neural networks and connect with complex brain networks responsible for motor control.
Researchers discovered that mismatch repair genes are critical in eliciting damages to neurons vulnerable to Huntington's disease, triggering downstream pathologies and motor impairment. Targeting these genes may offer novel therapeutic approaches, including improving locomotor and gait deficits and reducing neuronal cell death.
Research suggests that APOBEC enzymes, which normally target viruses, are unusually active in the brains of Huntington’s patients and cause genetic changes. The study found that APOBEC3A was most pronounced in causing DNA repeat expansion in a CAG/CTG tract.
Researchers found subtle brain changes in people with Huntington's disease 20 years before symptoms appear, offering hope for earlier interventions. The study identified early markers of neurodegeneration through advanced imaging and biomarkers.
A new study reveals that the inherited genetic mutation in Huntington’s disease doesn't harm cells immediately, but slowly morphs into a highly toxic form that kills the cell. The findings suggest potential ways to delay or even prevent the disease by stopping or slowing CAG-repeat expansion in the HTT gene.
An international team has presented the first detailed picture of Huntington's disease protein clumps, known as fibrils. These elongated shapes differ in important ways from those in other diseases like Alzheimer's and Parkinson's, offering new insights into their role in the disease.
Researchers at the University of Bergen have made a groundbreaking discovery in understanding the structure of protein clumps associated with Huntington's disease. The study provides new insights into the disease's mechanisms and paves the way for the development of diagnostic tools and treatments.
A new gene editing tool called SPLICER has been applied to reduce the formation of amyloid-beta plaque precursors in a mouse model of Alzheimer's disease. The application shows improved efficiency over current standard gene editing technology and potential for application in other diseases.
Researchers found that patients taking beta-blockers had a significantly lower risk of developing Huntington's symptoms and slower symptom worsening compared to non-users. This suggests that beta-blockers may provide benefit to patients at various stages of the disease.
A genetic mutation that causes Huntington's disease may also enhance early brain development and play a role in promoting human intelligence. Children with the HD mutation have bigger brains and higher IQ than children without the mutation.
Scientists have developed a polymer-based therapeutic for Huntington’s disease, which disrupts protein interactions to preserve cell health. The treatment successfully rescued neurons and reversed symptoms in mouse studies, showing promise as a potential delay or reduction of disease onset.
A study published in Nature Metabolism identifies a biochemical change responsible for Huntington's disease development and blocks disease progression by regulating dopamine levels. The research suggests that targeting an enzyme called GSTO2 could help prevent or delay the onset of motor symptoms.
The study analyzed the genetic profiles of 80,000 people and found that repeat expansion disorders (REDs) are common across different populations. The findings suggest a significant shift in how we think about genetic testing, profiling, and counseling for these conditions.
McMaster researchers found that the mutated huntingtin protein doesn't stimulate Poly [ADP-ribose] (PAR) production, resulting in less effective DNA repair. This discovery has implications for understanding Huntington’s Disease and potentially treating cancer.
Scientists have developed new therapies that selectively remove aggregated tau proteins associated with Alzheimer's disease in mice. The approach utilises TRIM21 to target tau aggregates, leaving healthy tau intact, and demonstrates potential for other brain disorders driven by protein aggregation.
A study published in Nature Communications implicates the gene CHCHD2 in Huntington's disease progression and identifies it as a potential therapeutic target. The researchers found that mutations in the HTT gene affect CHCHD2, which is involved in maintaining mitochondrial function.
Researchers discovered that Huntington's disease protein aggregates cause breaks in the nuclear envelope, leading to DNA damage and misregulation of neuronal genes. The study suggests a common mechanism for neurodegenerative diseases involving nuclear aggregate-induced ruptures.
A new study published in Nucleic Acid Therapeutics found that siRNA reduces huntingtin mRNA levels in the cytoplasm but not in the nucleus of mouse brains, suggesting a limitation in its effectiveness for treating Huntington's disease. The research highlights the importance of understanding the structure and function of nuclear RNA to ...
A new study by UCLA Health reveals racial disparities in Huntington's disease diagnoses, with Black patients receiving diagnoses one year later than White patients. The study analyzed nearly 5,000 patient data points and found that these disparities may exacerbate underrepresentation of minority groups in clinical trials.
Research finds that Huntington’s disease damages microscopic blood vessels in the brain, affecting coordination between neuronal activity and oxygenation. The study uses non-invasive measurement techniques to monitor disease progression and evaluate potential treatments.
Researchers have discovered that a rare type of lipid, with two polyunsaturated fatty acyl tails, promotes ferroptosis, a form of cell death. This finding could lead to new treatments for neurodegenerative diseases and induce cancer cell death.
The C-Path Neuroscience Annual Workshop brought together stakeholders to chart a transformative course for neurology research and drug development, focusing on chronic progressive diseases such as Alzheimer's and Parkinson's. Key highlights included recommendations for innovative therapies and tools to address complex disorders.
Researchers have identified a crucial biological trigger of Huntington's disease, finding that methylation converts an important protein into waste. By targeting this process, they may develop effective therapies for other neurodegenerative diseases.
A recent study published in Nature Medicine suggests that complement proteins and microglia can be activated early in the development of Huntington's Disease, leading to synapse loss and cognitive decline. By blocking these proteins, researchers were able to prevent or slow cognitive defects and motor symptoms in animal models.
Researchers at Chalmers University of Technology have shown that graphene oxide nanoflakes can reduce the accumulation of misfolded amyloid peptides in yeast cells, which are similar to human neurons affected by Alzheimer's disease. This suggests that graphene oxide may hold great potential for treating neurodegenerative diseases.
Researchers discovered a synthetic plant biology approach to prevent protein aggregation in human cells and nematodes, using the plant enzyme stromal processing peptidase (SPP) derived from chloroplasts. This finding opens the door to testing SPP as a potential therapy for Huntington's disease.
A new special issue of the Journal of Huntington’s Disease highlights the critical impact of sleep dysfunction on HD patients. Disrupted sleep can alter metabolism, increase vulnerability to infection, and exacerbate disease outcomes.
Scientists have discovered an additional source of genetic mutations that cause rare conditions like Huntington's disease. Expanded CAG repeat RNA can form aggregates that reduce global protein synthesis and lead to neurotoxicity.
Researchers at the University of Copenhagen have discovered a way to replace diseased and aged brain cells with new ones, which could lead to treatments for neurodegenerative diseases like Huntington's disease and multiple sclerosis. The study used humanized mice models to test the effectiveness of glial cell transplantation.
Researchers found that young and healthy human glial progenitor cells can outcompete older and diseased cells in the adult brain, replacing them with healthier ones. This breakthrough has strong therapeutic implications for treating neurological disorders like Huntington's disease.
Scientists found that cooling or warming the striatum region slows down or speeds up activity patterns, which correlates with rats' timing judgements. This provides evidence for the 'population clock hypothesis', suggesting that brains use decentralized and flexible sense of time.
A new study describes an engineered approach that makes protein aggregates amenable to spatial manipulations in both budding yeast and human cells. This system allows for the export of protein aggregates from cells, potentially protecting mother cells from toxicity and contributing to a better understanding of neurodegenerative diseases.
Researchers created a detailed 3D image of the synapse, a key juncture in neuronal communication. The model reveals the precise geometry of interactions between individual cells, which may hold the key to understanding neurodegenerative diseases.
Scientists at the Stowers Institute for Medical Research have uncovered the structure of the first step in amyloid formation for Huntington's disease. The team proposes a new method for treating not only Huntington's but potentially dozens of other amyloid-associated diseases by preventing the initial, rate-limiting step from occurring.
Research by West Virginia University professor Sean Tu found that orphan drugs earn pharmaceutical companies almost as much as those marketed to the general public. The Orphan Drug Act incentivizes companies with tax credits, longer patent exclusivity, and easier FDA review for treating rare diseases.
A study published in the Journal of Neuroscience has identified new alterations in neural circuits in mice models of Huntington's disease, significantly impacting its lives. The research found that the M2 cortex sends axonal projections to the superior colliculus, which are deeply impaired and linked to disease symptomatology.
A University of Ottawa team has discovered a vital role for the VGLUT3 transporter protein in modulating the development of Huntington's disease. The study shows that blocking glutamate release through this protein can lead to an amelioration of the disease progression, offering new hope for potential treatment approaches.