NCBI-Aggregator

Profiles of patients at early stages of Huntington's disease based on the routine biological markers and the disease progression

J Neurol. 2026 Apr 2;273(4):249. doi: 10.1007/s00415-026-13717-0.ABSTRACTBACKGROUND: Huntington's disease (HD) is a neurodegenerative disorder characterized by motor, cognitive, and psychiatric symptoms. Many studies attempt, besides age and CAG, to understand the factors that impact disease progression. Biological tests measuring several metabolic and inflammatory factors are frequently performed in outpatients, but...Read more

Source: NCBI Date: 2026-04-02 By Andres Gil-Salcedo
Inhibition of the NLRP3 Inflammasome With MCC950 Improves Gut Health in Huntington's Disease Mice

J Neurochem. 2026 Apr;170(4):e70419. doi: 10.1111/jnc.70419.ABSTRACTHuntington's disease (HD) is an autosomal dominant neurodegenerative disorder featuring abnormal cognition, psychiatric symptoms, movement, and gastrointestinal function. It is caused by a tandem-repeat gene mutation encoding an expanded polyglutamine tract in the huntingtin protein. Our group was the first to demonstrate gut microbial disruption...Read more

Source: NCBI Date: 2026-03-30 By Sujan Kumar Sarkar
Clozapine-Induced Myocarditis in Huntington's Disease: Case Report

Turk Psikiyatri Derg. 2026;37:49-53. doi: 10.5080/u27742.ABSTRACTHuntington’s disease (HD) is an autosomal dominant neurodegenerative disease. Its clinical presentation is primarily characterized by motor dysfunction, cognitive decline, and a broad spectrum of neuropsychiatric symptoms. Common psychiatric manifestations of HD include mood and anxiety disorders as well as behavioral and personality changes. Psychosis...Read more

Source: NCBI Date: 2026-03-27 By Nur Özgedik Turhan
A small-molecule stabilizer of the calpastatin-calpain-2 complex restores mitochondrial function and mitigates neurodegeneration

Sci Adv. 2026 Mar 27;12(13):eaeb1174. doi: 10.1126/sciadv.aeb1174. Epub 2026 Mar 27.ABSTRACTMitochondrial dysfunction and dysregulated proteolysis drive Huntington's disease (HD), tauopathy, and related neurodegenerative disorders. Calpain-2, a Ca2+-activated protease restrained by calpastatin (CAST), is pathologically overactivated, yet no therapies directly target this axis. We identify A36, a brain-penetrant small molecule derived...Read more

Source: NCBI Date: 2026-03-27 By Di Hu
Selective targeting of mutant huntingtin intron 1 improves rescue provided by antisense oligonucleotides in Huntington's disease mice

Sci Transl Med. 2026 Mar 18;18(841):eadv0702. doi: 10.1126/scitranslmed.adv0702. Epub 2026 Mar 18.ABSTRACTHuntington's disease (HD) arises from the toxic gain of function caused by a CAG expansion in the coding region of the huntingtin (HTT) gene. HD is increasingly appreciated to emerge from multiple pathogenic processes, including somatic instability in mutant...Read more

Source: NCBI Date: 2026-03-18 By Robert M Bragg
Lowering the HTT1a transcript as an effective therapy for Huntington's disease in a knockin mouse model

Sci Transl Med. 2026 Mar 18;18(841):eadw2495. doi: 10.1126/scitranslmed.adw2495. Epub 2026 Mar 18.ABSTRACTLowering huntingtin (HTT) transcript levels has been a major focus of therapeutic development for Huntington's disease (HD), but which transcript should be lowered? HD is caused by a CAG repeat expansion in exon 1 of the HTT gene, and...Read more

Source: NCBI Date: 2026-03-18 By Aikaterini Smaragdi Papadopoulou
Self-inactivating AAV-CRISPR at different ages enables sustained amelioration of Huntington's disease deficits in BAC226Q mice

Sci Adv. 2026 Mar 20;12(12):eaea8052. doi: 10.1126/sciadv.aea8052. Epub 2026 Mar 18.ABSTRACTHuntington's disease (HD) is a monogenic autosomal dominant neurodegenerative disorder caused by a CAG repeat expansion in exon 1 of the HTT gene, yielding a gain-of-toxic-function mutant Huntingtin protein (mHTT). CRISPR-Cas9 is a potentially powerful therapeutic strategy for HD by...Read more

Source: NCBI Date: 2026-03-18 By Yuanyi Dai
Human iPSC-derived exosomes for amelioration of Huntington's disease through mitochondrial, synaptic, and anti-apoptotic mechanisms

Biomed Pharmacother. 2026 Apr;197:119217. doi: 10.1016/j.biopha.2026.119217. Epub 2026 Mar 16.ABSTRACTHuntington's disease (HD) is driven by expanded CAG repeats in the huntingtin gene, resulting in mutant huntingtin (mHtt) aggregation, mitochondrial dysfunction, neuronal loss, and neuroinflammation. Although stem cell-based therapies provide potent regenerative, anti-inflammatory, and neuroprotective effects, their clinical translation remains constrained...Read more

Source: NCBI Date: 2026-03-18 By Seoho Jahng
Discovery and Optimization of Thienopyrazine RNA-Splicing Modulators for the Treatment of Huntington's Disease

J Med Chem. 2026 Mar 26;69(6):7427-7442. doi: 10.1021/acs.jmedchem.6c00224. Epub 2026 Mar 17.ABSTRACTHuntington's disease (HD) is a progressive neurodegenerative disorder caused by a CAG-repeat expansion in the Huntington gene (HTT). Herein, we describe the discovery of a series of HTT pre-mRNA-splicing modulators that promote the inclusion of a cryptic stop codon...Read more

Source: NCBI Date: 2026-03-17 By Chaofan Xu
Mitochondria "Shackled" by Mutant Huntingtin: Analysis of Morphological Alterations and Disruptions of Intracellular Transport

Biochemistry (Mosc). 2026 Feb;91(2):253-273. doi: 10.1134/S0006297925602850.ABSTRACTMitochondria are semi-autonomous, multifunctional organelles that supply cells with energy. They are highly dynamic structures, capable of moving, fusing, dividing, and forming branched networks. The number, density, and complexity of mitochondrial network are unique to each cell type and reflect cellular demands for ATP and...Read more

Source: NCBI Date: 2026-03-17 By Vyacheslav I Pasko
A Two-Track Model of Huntington's Disease Pathology: Striatal Atrophy Mediates Maladaptive Immune Dysregulation

Int J Mol Sci. 2026 Mar 4;27(5):2384. doi: 10.3390/ijms27052384.ABSTRACTHuntington's disease (HD) is characterized by progressive striatal atrophy and complex proteomic changes in the central nervous system. Using the ultrasensitive Next-Gen Ultra-Sensitive Immunoassay (NULISA) proteomic platform, we analyzed cerebrospinal fluid (CSF) from 88 persons with HD to dissect the biological correlates...Read more

Source: NCBI Date: 2026-03-14 By H Jeremy Bockholt
Huntington's disease LIG1 modifier variant increases ligase fidelity and suppresses somatic CAG repeat expansion

Proc Natl Acad Sci U S A. 2026 Mar 10;123(10):e2518854123. doi: 10.1073/pnas.2518854123. Epub 2026 Mar 2.ABSTRACTHuntington's disease (HD) is a fatal neurodegenerative disorder caused by inheriting an expanded CAG repeat tract in the huntingtin gene (HTT) that further expands in somatic cells over an individual's lifetime. Genome-wide association studies have...Read more

Source: NCBI Date: 2026-03-01 By Eunhye Lee
Surface-Engineered Precision Nano-Systems for Targeted Treatment of Huntington's Disease: A Review of Recent Advancements

Int J Nanomedicine. 2026 Feb 20;21:548892. doi: 10.2147/IJN.S548892. eCollection 2026.ABSTRACTHuntington's disease is a progressive neurological disorder marked by motor, cognitive, and psychiatric symptoms. Currently, there are no definitive diagnostic tools or effective treatments to halt or reverse the disease. In recent years, surface-engineered nanosystems have emerged as innovative therapeutic platforms,...Read more

Source: NCBI Date: 2026-02-26 By Jingze Zhang
Electroencephalography biomarkers in Huntington's disease: A systematic review of resting-state and sleep EEG alterations

Clin Neurophysiol. 2026 May;185:2111701. doi: 10.1016/j.clinph.2026.2111701. Epub 2026 Feb 12.ABSTRACTOBJECTIVE: To systematically review EEG-based biomarkers in Huntington's disease across resting-state, sleep, medication response, and clinical correlation studies.METHODS: PubMed, Web of Science, and EMBASE databases were systematically searched for studies of sufficient methodological quality that examined EEG alterations in patients with...Read more

Source: NCBI Date: 2026-02-25 By Sayeh Jalali
Silmitasertib, an FDA-designated orphan CK2 inhibitor, ameliorates neuropathology and motor dysfunction in a Huntington's disease mouse model

Neurotherapeutics. 2026 Jan;23(1):e00859. doi: 10.1016/j.neurot.2026.e00859. Epub 2026 Feb 24.ABSTRACTHuntington's disease (HD) is a devastating autosomal dominant neurodegenerative disease that manifests with progressive motor, cognitive, and psychological impairments. HD is caused by a CAG (glutamine) repeat expansion in the huntingtin (HTT) gene, leading to the misfolding and aggregation of mutant HTT...Read more

Source: NCBI Date: 2026-02-24 By Ross J Pelzel

…