Therapeutic approaches to Huntington disease: from the bench to the clinic

Estimated number of off-target candidate sites for antisense oligonucleotides in human mRNA sequences

(2018) Tokuyuki Yoshida et al.   GENES TO CELLS

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

(2018) Edith L. Pfister et al.   HUMAN GENE THERAPY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Improved metrics for Huntington’s disease trials

(2018) Kat Arney   NATURE

Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain

(2018) Yasunori Matsuzaki et al.   NEUROSCIENCE LETTERS

CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease

(2017) Su Yang et al.   JOURNAL OF CLINICAL INVESTIGATION

Neurofilament light protein in blood as a potential biomarker of neurodegeneration in Huntington's disease: a retrospective cohort analysis

(2017) Lauren M Byrne et al.   LANCET NEUROLOGY

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

(2017) Alex Mas Monteys et al.   MOLECULAR THERAPY

Neurotoxic reactive astrocytes are induced by activated microglia

(2017) Shane A. Liddelow et al.   NATURE

The sigma-1 receptor mediates the beneficial effects of pridopidine in a mouse model of Huntington disease

(2017) Daniel Ryskamp et al.   NEUROBIOLOGY OF DISEASE

Motor, cognitive, and functional declines contribute to a single progressive factor in early HD

(2017) Scott A. Schobel et al.   NEUROLOGY

The CREST-E study of creatine for Huntington disease

(2017) Steven M. Hersch et al.   NEUROLOGY

Phosphorylation of huntingtin at residue T3 is decreased in Huntington’s disease and modulates mutant huntingtin protein conformation

(2017) Cristina Cariulo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Huntington’s Disease iPSC-Derived Brain Microvascular Endothelial Cells Reveal WNT-Mediated Angiogenic and Blood-Brain Barrier Deficits

(2017) Ryan G. Lim et al.   Cell Reports

The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes

(2017) Nicolas Merienne et al.   Cell Reports

Elimination of huntingtin in the adult mouse leads to progressive behavioral deficits, bilateral thalamic calcification, and altered brain iron homeostasis

(2017) Paula Dietrich et al.   PLoS Genetics

Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells

(2017) Xiaohong Xu et al.   Stem Cell Reports

Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing

(2017) Karine Cambon et al.   Molecular Therapy-Methods & Clinical Development

Bupropion for the treatment of apathy in Huntington’s disease: A multicenter, randomised, double-blind, placebo-controlled, prospective crossover trial

(2017) Harald Gelderblom et al.   PLoS One

The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain

(2017) Nicole A. Datson et al.   PLoS One

Impairment of blood-brain barrier is an early event in R6/2 mouse model of Huntington Disease

(2017) Alba Di Pardo et al.   Scientific Reports

The pathogenic exon 1 HTT protein is produced by incomplete splicing in Huntington’s disease patients

(2017) Andreas Neueder et al.   Scientific Reports

Globus pallidus degeneration and clinicopathological features of Huntington disease

(2016) Malvindar K. Singh-Bains et al.   ANNALS OF NEUROLOGY

In vivo proof-of-concept of removal of the huntingtin caspase cleavage motif-encoding exon 12 approach in the YAC128 mouse model of Huntington’s disease

(2016) João Casaca-Carreira et al.   BIOMEDICINE & PHARMACOTHERAPY

The targetable A1 Huntington disease haplotype has distinct Amerindian and European origins in Latin America

(2016) Chris Kay et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

A novel neurodevelopmental disorder associated with compound heterozygous variants in the huntingtin gene

(2016) Lance H Rodan et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

The novel KMO inhibitor CHDI-340246 leads to a restoration of electrophysiological alterations in mouse models of Huntington's disease

(2016) Vahri Beaumont et al.   EXPERIMENTAL NEUROLOGY

Treatment with the MAO-A inhibitor clorgyline elevates monoamine neurotransmitter levels and improves affective phenotypes in a mouse model of Huntington disease

(2016) Marta Garcia-Miralles et al.   EXPERIMENTAL NEUROLOGY

Laquinimod decreases Bax expression and reduces caspase-6 activation in neurons

(2016) Dagmar E. Ehrnhoefer et al.   EXPERIMENTAL NEUROLOGY

Effect of Deutetrabenazine on Chorea Among Patients With Huntington Disease

(2016) et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Serine 421 regulates mutant huntingtin toxicity and clearance in mice

(2016) Ian H. Kratter et al.   JOURNAL OF CLINICAL INVESTIGATION

Laquinimod dampens hyperactive cytokine production in Huntington's disease patient myeloid cells

(2016) Lucianne Dobson et al.   JOURNAL OF NEUROCHEMISTRY

A double-blind, randomized, cross-over, placebo-controlled, pilot trial with Sativex in Huntington’s disease

(2016) Jose Luis López-Sendón Moreno et al.   JOURNAL OF NEUROLOGY

Functional Indicators of Glutamate Transport in Single Striatal Astrocytes and the Influence of Kir4.1 in Normal and Huntington Mice

(2016) Anton Dvorzhak et al.   JOURNAL OF NEUROSCIENCE

Mutant Huntingtin Impairs BDNF Release from Astrocytes by Disrupting Conversion of Rab3a-GTP into Rab3a-GDP

(2016) Y. Hong et al.   JOURNAL OF NEUROSCIENCE

Dysfunctional Calcium and Glutamate Signaling in Striatal Astrocytes from Huntington's Disease Model Mice

(2016) R. Jiang et al.   JOURNAL OF NEUROSCIENCE

Symptom heterogeneity in Huntington's disease correlates with neuronal degeneration in the cerebral cortex

(2016) Nasim F. Mehrabi et al.   NEUROBIOLOGY OF DISEASE

A randomized, double-blind, placebo-controlled trial of coenzyme Q10 in Huntington disease

(2016) Andrew McGarry et al.   NEUROLOGY

Phosphodiesterase 10A Inhibition Improves Cortico-Basal Ganglia Function in Huntington’s Disease Models

(2016) Vahri Beaumont et al.   NEURON

Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis

(2016) Guohao Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Deutetrabenazine—Not a Revolution but Welcome Evolution for Treating Chorea in Huntington Disease

(2016) Ralf Reilmann   JAMA Neurology

Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease

(2016) Piotr Hadaczek et al.   Molecular Therapy-Methods & Clinical Development

Laquinimod rescues striatal, cortical and white matter pathology and results in modest behavioural improvements in the YAC128 model of Huntington disease

(2016) Marta Garcia-Miralles et al.   Scientific Reports

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

An exploratory double-blind, randomized clinical trial with selisistat, a SirT1 inhibitor, in patients with Huntington's disease

(2015) Sigurd D. Süssmuth et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Effect of Creatine Monohydrate on Clinical Progression in Patients With Parkinson Disease

(2015) Karl Kieburtz et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Pridopidine, a dopamine stabilizer, improves motor performance and shows neuroprotective effects in Huntington disease R6/2 mouse model

(2015) Ferdinando Squitieri et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington’s disease patients

(2015) Edward J. Wild et al.   JOURNAL OF CLINICAL INVESTIGATION

Deep brain stimulation of the internal pallidum in Huntington’s disease patients: clinical outcome and neuronal firing patterns

(2015) Cécile Delorme et al.   JOURNAL OF NEUROLOGY

Safety, tolerability, and efficacy of PBT2 in Huntington's disease: a phase 2, randomised, double-blind, placebo-controlled trial

(2015)   LANCET NEUROLOGY

Nonclinical Safety Evaluation of VX15/2503, a Humanized IgG4 Anti-SEMA4D Antibody

(2015) J. E. Leonard et al.   MOLECULAR CANCER THERAPEUTICS

A randomized, placebo-controlled trial of AFQ056 for the treatment of chorea in Huntington's disease

(2015) Ralf Reilmann et al.   MOVEMENT DISORDERS

SEMA4D compromises blood–brain barrier, activates microglia, and inhibits remyelination in neurodegenerative disease

(2015) Ernest S. Smith et al.   NEUROBIOLOGY OF DISEASE

Anti-semaphorin 4D immunotherapy ameliorates neuropathology and some cognitive impairment in the YAC128 mouse model of Huntington disease

(2015) Amber L. Southwell et al.   NEUROBIOLOGY OF DISEASE

Mutant Huntingtin Downregulates Myelin Regulatory Factor-Mediated Myelin Gene Expression and Affects Mature Oligodendrocytes

(2015) Brenda Huang et al.   NEURON

RAN Translation in Huntington Disease

(2015) Monica Bañez-Coronel et al.   NEURON

Pallidal deep brain stimulation in Huntington's disease

(2015) S. Zittel et al.   PARKINSONISM & RELATED DISORDERS

Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression

(2015) Amber L. Southwell et al.   Scientific Reports

Feasibility of Virtual Research Visits in Fox Trial Finder

(2015) E. Ray Dorsey et al.   Journal of Parkinsons Disease

Novel Methods and Technologies for 21st-Century Clinical Trials

(2015) E. Ray Dorsey et al.   JAMA Neurology

A Prospective Pilot Trial for Pallidal Deep Brain Stimulation in Huntington’s Disease

(2015) Lars Wojtecki et al.   Frontiers in Neurology

Cortical interneuron loss and symptom heterogeneity in Huntington disease

(2014) Eric H. Kim et al.   ANNALS OF NEUROLOGY

Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease

(2014) Lisa M. Stanek et al.   HUMAN GENE THERAPY

Identification of a post-translationally myristoylated autophagy-inducing domain released by caspase cleavage of Huntingtin

(2014) D. D. O. Martin et al.   HUMAN MOLECULAR GENETICS

Deep brain stimulation for Huntington's disease: long-term results of a prospective open-label study

(2014) Victoria Gonzalez et al.   JOURNAL OF NEUROSURGERY

Inhibition of Excessive Monoamine Oxidase A/B Activity Protects Against Stress-induced Neuronal Death in Huntington Disease

(2014) Jolene Ooi et al.   MOLECULAR NEUROBIOLOGY

In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

(2014) Amber L Southwell et al.   MOLECULAR THERAPY

Intrajugular Vein Delivery of AAV9-RNAi Prevents Neuropathological Changes and Weight Loss in Huntington's Disease Mice

(2014) Brett D Dufour et al.   MOLECULAR THERAPY

Prospects for neuroprotective therapies in prodromal Huntington's disease

(2014) Abhishek Chandra et al.   MOVEMENT DISORDERS

Diagnostic criteria for Huntington's disease based on natural history

(2014) Ralf Reilmann et al.   MOVEMENT DISORDERS

A practical approach to remote longitudinal follow-up of Parkinson's disease: The FOUND study

(2014) Caroline M. Tanner et al.   MOVEMENT DISORDERS

Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease

(2014) Nan Wang et al.   NATURE MEDICINE

Mutant Huntingtin promotes autonomous microglia activation via myeloid lineage-determining factors

(2014) Andrea Crotti et al.   NATURE NEUROSCIENCE

Astrocyte Kir4.1 ion channel deficits contribute to neuronal dysfunction in Huntington's disease model mice

(2014) Xiaoping Tong et al.   NATURE NEUROSCIENCE

Huntington disease: natural history, biomarkers and prospects for therapeutics

(2014) Christopher A. Ross et al.   Nature Reviews Neurology

PRECREST: A phase II prevention and biomarker trial of creatine in at-risk Huntington disease

(2014) H. D. Rosas et al.   NEUROLOGY

Phosphorylation of Mutant Huntingtin at Serine 116 Modulates Neuronal Toxicity

(2014) Erin E. Watkin et al.   PLoS One

Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

(2014) Niels H. Skotte et al.   PLoS One

Preventing Formation of Toxic N-Terminal Huntingtin Fragments Through Antisense Oligonucleotide-Mediated Protein Modification

(2014) Melvin M. Evers et al.   Nucleic Acid Therapeutics

The Phosphodiesterase 10 Positron Emission Tomography Tracer, [18F]MNI-659, as a Novel Biomarker for Early Huntington Disease

(2014) David S. Russell et al.   JAMA Neurology

Laquinimod reduces neuroaxonal injury through inhibiting microglial activation

(2014) Manoj Kumar Mishra et al.   Annals of Clinical and Translational Neurology

Huntington disease in the South African population occurs on diverse and ethnically distinct genetic haplotypes

(2013) Fiona K Baine et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

PDE10 inhibition increases GluA1 and CREB phosphorylation and improves spatial and recognition memories in a Huntington's disease mouse model

(2013) Albert Giralt et al.   HIPPOCAMPUS

The huntingtin N17 domain is a multifunctional CRM1 and Ran-dependent nuclear and cilial export signal

(2013) T. Maiuri et al.   HUMAN MOLECULAR GENETICS

Live cell imaging and biophotonic methods reveal two types of mutant huntingtin inclusions

(2013) Nicholas S. Caron et al.   HUMAN MOLECULAR GENETICS

Prevalence of adult Huntington's disease in the UK based on diagnoses recorded in general practice records

(2013) Stephen JW Evans et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data

(2013) Sarah J Tabrizi et al.   LANCET NEUROLOGY

A randomized, double-blind, placebo-controlled trial of pridopidine in Huntington's disease

(2013)   MOVEMENT DISORDERS

Multisource ascertainment of Huntington disease in Canada: Prevalence and population at risk

(2013) Emily R. Fisher et al.   MOVEMENT DISORDERS

Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon

(2013) Melvin M. Evers et al.   NEUROBIOLOGY OF DISEASE

Chronic blockade of extrasynaptic NMDA receptors ameliorates synaptic dysfunction and pro-death signaling in Huntington disease transgenic mice

(2013) Alejandro Dau et al.   NEUROBIOLOGY OF DISEASE

PET imaging shows loss of striatal PDE10A in patients with Huntington disease

(2013) R. Ahmad et al.   NEUROLOGY

One-year safety and tolerability profile of pridopidine in patients with Huntington disease

(2013) F. Squitieri et al.   NEUROLOGY

Polyglutamine domain flexibility mediates the proximity between flanking sequences in huntingtin

(2013) Nicholas Stephane Caron et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease

(2013) K. Sathasivam et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Sigma-1 receptor agonist PRE084 is protective against mutant huntingtin-induced cell degeneration: involvement of calpastatin and the NF-κB pathway

(2013) A Hyrskyluoto et al.   Cell Death & Disease

Soluble forms of polyQ-expanded huntingtin rather than large aggregates cause endoplasmic reticulum stress

(2013) Julia Leitman et al.   Nature Communications

Reduced astrocytic NF-κB activation by laquinimod protects from cuprizone-induced demyelination

(2012) Wolfgang Brück et al.   ACTA NEUROPATHOLOGICA

Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum

(2012) Richard Grondin et al.   BRAIN

Axonal transport of adeno-associated viral vectors is serotype-dependent

(2012) E A Salegio et al.   GENE THERAPY

Mutant huntingtin impairs immune cell migration in Huntington disease

(2012) Wanda Kwan et al.   JOURNAL OF CLINICAL INVESTIGATION

Oral treatment with laquinimod augments regulatory T-cells and brain-derived neurotrophic factor expression and reduces injury in the CNS of mice with experimental autoimmune encephalomyelitis

(2012) Rina Aharoni et al.   JOURNAL OF NEUROIMMUNOLOGY

The dopamine stabilizers ACR16 and (−)-OSU6162 display nanomolar affinities at the σ-1 receptor

(2012) K Sahlholm et al.   MOLECULAR PSYCHIATRY

Striatal neuronal loss correlates with clinical motor impairment in Huntington's disease

(2012) Zhihong Guo et al.   MOVEMENT DISORDERS

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

Ganglioside GM1 induces phosphorylation of mutant huntingtin and restores normal motor behavior in Huntington disease mice

(2012) A. Di Pardo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice

(2012) M. Garriga-Canut et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases

(2012) Amber L. Southwell et al.   TRENDS IN MOLECULAR MEDICINE

A Pathogenic Mechanism in Huntington's Disease Involves Small CAG-Repeated RNAs with Neurotoxic Activity

(2012) Mónica Bañez-Coronel et al.   PLoS Genetics

Modulation of Autoimmune Demyelination by Laquinimod via Induction of Brain-Derived Neurotrophic Factor

(2011) Jan Thöne et al.   AMERICAN JOURNAL OF PATHOLOGY

Kynurenine 3-Monooxygenase Inhibition in Blood Ameliorates Neurodegeneration

(2011) Daniel Zwilling et al.   CELL

The Kynurenine Pathway Modulates Neurodegeneration in a Drosophila Model of Huntington's Disease

(2011) Susanna Campesan et al.   CURRENT BIOLOGY

HTT haplotypes contribute to differences in Huntington disease prevalence between Europe and East Asia

(2011) Simon C Warby et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Protein aggregates in Huntington's disease

(2011) Montserrat Arrasate et al.   EXPERIMENTAL NEUROLOGY

Mutant huntingtin causes defective actin remodeling during stress: defining a new role for transglutaminase 2 in neurodegenerative disease

(2011) Lise Munsie et al.   HUMAN MOLECULAR GENETICS

Pridopidine for the treatment of motor function in patients with Huntington's disease (MermaiHD): a phase 3, randomised, double-blind, placebo-controlled trial

(2011) Justo Garcia de Yebenes et al.   LANCET NEUROLOGY

Potential endpoints for clinical trials in premanifest and early Huntington's disease in the TRACK-HD study: analysis of 24 month observational data

(2011) Sarah J Tabrizi et al.   LANCET NEUROLOGY

Mass Spectrometric Identification of Novel Lysine Acetylation Sites in Huntingtin

(2011) Xin Cong et al.   MOLECULAR & CELLULAR PROTEOMICS

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

(2011) Jeffrey B Carroll et al.   MOLECULAR THERAPY

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

(2011) Jodi L McBride et al.   MOLECULAR THERAPY

Kinase inhibitors modulate huntingtin cell localization and toxicity

(2011) Randy Singh Atwal et al.   Nature Chemical Biology

Mutant huntingtin binds the mitochondrial fission GTPase dynamin-related protein-1 and increases its enzymatic activity

(2011) Wenjun Song et al.   NATURE MEDICINE

Convergent pathogenic pathways in Alzheimer's and Huntington's diseases: shared targets for drug development

(2011) Dagmar E. Ehrnhoefer et al.   NATURE REVIEWS DRUG DISCOVERY

Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide

(2011) Melvin M. Evers et al.   PLoS One

Allele-Selective Inhibition of MutantHuntingtinExpression with Antisense Oligonucleotides Targeting the Expanded CAG Repeat

(2010) Keith T. Gagnon et al.   BIOCHEMISTRY

Cell loss in the motor and cingulate cortex correlates with symptomatology in Huntington’s disease

(2010) Doris C. V. Thu et al.   BRAIN

Allele-Selective Inhibition of Huntingtin Expression by Switching to an miRNA-like RNAi Mechanism

(2010) Jiaxin Hu et al.   CHEMISTRY & BIOLOGY

In vivo expression of polyglutamine-expanded huntingtin by mouse striatal astrocytes impairs glutamate transport: a correlation with Huntington's disease subjects

(2010) M. Faideau et al.   HUMAN MOLECULAR GENETICS

Longitudinal change in regional brain volumes in prodromal Huntington disease

(2010) E. H. Aylward et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Cleavage at the 586 Amino Acid Caspase-6 Site in Mutant huntingtin Influences Caspase-6 Activation In Vivo

(2010) R. K. Graham et al.   JOURNAL OF NEUROSCIENCE

Alzheimer's disease: clinical trials and drug development

(2010) Francesca Mangialasche et al.   LANCET NEUROLOGY

Biological and clinical changes in premanifest and early stage Huntington's disease in the TRACK-HD study: the 12-month longitudinal analysis

(2010) Sarah J Tabrizi et al.   LANCET NEUROLOGY

Location, Location, Location: Contrasting Roles of Synaptic and Extrasynaptic NMDA Receptors in Huntington's Disease

(2010) Michael S. Levine et al.   NEURON

Early Increase in Extrasynaptic NMDA Receptor Signaling and Expression Contributes to Phenotype Onset in Huntington's Disease Mice

(2010) Austen J. Milnerwood et al.   NEURON

Inhibition of the Striatal Specific Phosphodiesterase PDE10A Ameliorates Striatal and Cortical Pathology in R6/2 Mouse Model of Huntington's Disease

(2010) Carmela Giampà et al.   PLoS One

Molecular mechanisms of excitotoxicity and their relevance to pathogenesis of neurodegenerative diseases

(2009) Xiao-xia Dong et al.   ACTA PHARMACOLOGICA SINICA

CAG Expansion in the Huntington Disease Gene Is Associated with a Specific and Targetable Predisposing Haplogroup

(2009) Simon C. Warby et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Sustained effects of nonallele-specificHuntingtinsilencing

(2009) Valérie Drouet et al.   ANNALS OF NEUROLOGY

Prevention of depressive behaviour in the YAC128 mouse model of Huntington disease by mutation at residue 586 of huntingtin

(2009) M. A. Pouladi et al.   BRAIN

Acetylation Targets Mutant Huntingtin to Autophagosomes for Degradation

(2009) Hyunkyung Jeong et al.   CELL

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

(2009) Edith L. Pfister et al.   CURRENT BIOLOGY

The dopaminergic stabilizers pridopidine (ACR16) and (−)-OSU6162 display dopamine D2 receptor antagonism and fast receptor dissociation properties

(2009) Tino Dyhring et al.   EUROPEAN JOURNAL OF PHARMACOLOGY

Huntingtin facilitates polycomb repressive complex 2

(2009) Ihn Sik Seong et al.   HUMAN MOLECULAR GENETICS

Phosphorylation of Threonine 3

(2009) Charity T. Aiken et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

IKK phosphorylates Huntingtin and targets it for degradation by the proteasome and lysosome

(2009) Leslie Michels Thompson et al.   JOURNAL OF CELL BIOLOGY

Roles of Sema4D-Plexin-B1 Interactions in the Central Nervous System for Pathogenesis of Experimental Autoimmune Encephalomyelitis

(2009) T. Okuno et al.   JOURNAL OF IMMUNOLOGY

Interaction of Postsynaptic Density Protein-95 with NMDA Receptors Influences Excitotoxicity in the Yeast Artificial Chromosome Mouse Model of Huntington's Disease

(2009) J. Fan et al.   JOURNAL OF NEUROSCIENCE

Beyond the brain: widespread pathology in Huntington's disease

(2009) Jorien MM van der Burg et al.   LANCET NEUROLOGY

Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data

(2009) Sarah J Tabrizi et al.   LANCET NEUROLOGY

Mutant Huntingtin Impairs Vesicle Formation from Recycling Endosomes by Interfering with Rab11 Activity

(2009) X. Li et al.   MOLECULAR AND CELLULAR BIOLOGY

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

(2009) Ryan L Boudreau et al.   MOLECULAR THERAPY

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs

(2009) Jiaxin Hu et al.   NATURE BIOTECHNOLOGY

Balance between synaptic versus extrasynaptic NMDA receptor activity influences inclusions and neurotoxicity of mutant huntingtin

(2009) Shu-ichi Okamoto et al.   NATURE MEDICINE

Serines 13 and 16 Are Critical Determinants of Full-Length Human Mutant Huntingtin Induced Disease Pathogenesis in HD Mice

(2009) Xiaofeng Gu et al.   NEURON

Distinct neuroinflammatory profile in post-mortem human Huntingtonʼs disease

(2009) Aurelio Silvestroni et al.   NEUROREPORT

Expression of mutant huntingtin in mouse brain astrocytes causes age-dependent neurological symptoms

(2009) Jennifer Bradford et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells

(2009) D. Mittelman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Cerebral cortex and the clinical expression of Huntington's disease: complexity and heterogeneity

(2008) H. D. Rosas et al.   BRAIN

Neuronal viability is controlled by a functional relation between synaptic and extrasynaptic NMDA receptors

(2008) F. Léveillé et al.   FASEB JOURNAL

Identification and Allele-Specific Silencing of the Mutant Huntingtin Allele in Huntington's Disease Patient-Derived Fibroblasts

(2008) P.H.J. van Bilsen et al.   HUMAN GENE THERAPY

A novel pathogenic pathway of immune activation detectable before clinical onset in Huntington's disease

(2008) Maria Björkqvist et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Allele-specific silencing of mutant Huntington’s disease gene

(2008) Yu Zhang et al.   JOURNAL OF NEUROCHEMISTRY

N-Terminal Mutant Huntingtin Associates with Mitochondria and Impairs Mitochondrial Trafficking

(2008) A. L. Orr et al.   JOURNAL OF NEUROSCIENCE

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

(2008) M. Penaud-Budloo et al.   JOURNAL OF VIROLOGY

AAV Vector–mediated RNAi of Mutant Huntingtin Expression Is Neuroprotective in a Novel Genetic Rat Model of Huntington's Disease

(2008) Nicholas R Franich et al.   MOLECULAR THERAPY

Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): Scale presentation and clinimetric testing results

(2008) Christopher G. Goetz et al.   MOVEMENT DISORDERS

Synaptic NMDA receptor activity boosts intrinsic antioxidant defenses

(2008) Sofia Papadia et al.   NATURE NEUROSCIENCE

Symptomatic treatment of Huntington disease

(2008) Octavian R. Adam et al.   Neurotherapeutics

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

(2008) J. L. McBride et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Detection of Huntington's disease decades before diagnosis: the Predict-HD study

(2007) J S Paulsen et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY