Huntingtin Lowering Strategies

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality

(2019) Eloise Hudry et al.   NEURON

Huntingtin Lowering Strategies for Disease Modification in Huntington’s Disease

(2019) Sarah J. Tabrizi et al.   NEURON

Targeting Huntingtin Expression in Patients with Huntington’s Disease

(2019) Sarah J. Tabrizi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Prevalence of Carriers of Intermediate and Pathological Polyglutamine Disease–Associated Alleles Among Large Population-Based Cohorts

(2019) Sarah L. Gardiner et al.   JAMA Neurology

Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

(2019) Bryan Zeitler et al.   NATURE MEDICINE

CAG Repeat Not Polyglutamine Length Determines Timing of Huntington’s Disease Onset

(2019) Jong-Min Lee et al.   CELL

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

(2019) Julia F. Alterman et al.   NATURE BIOTECHNOLOGY

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

(2019) Jinkuk Kim et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Comprehensive Haplotype-Targeting Strategy for Allele-Specific HTT Suppression in Huntington Disease

(2019) Chris Kay et al.   AMERICAN JOURNAL OF HUMAN GENETICS

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

(2019) Sonay Keskin et al.   Molecular Therapy-Methods & Clinical Development

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Evaluation of mutant huntingtin and neurofilament proteins as potential markers in Huntington’s disease

(2018) Lauren M. Byrne et al.   Science Translational Medicine

Huntington's disease: a clinical review

(2017) P. McColgan et al.   EUROPEAN JOURNAL OF NEUROLOGY

Contribution of Neuroepigenetics to Huntington’s Disease

(2017) Laetitia Francelle et al.   Frontiers in Human Neuroscience

Antisense oligonucleotides: the next frontier for treatment of neurological disorders

(2017) Carlo Rinaldi et al.   Nature Reviews Neurology

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

(2017) Scott A. Schobel et al.   NEUROLOGY

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Endoplasmic reticulum stress: The cause and solution to Huntington's disease?

(2016) Yuwei Jiang et al.   BRAIN RESEARCH

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

Neuroimmunology of Huntington’s Disease: Revisiting Evidence from Human Studies

(2016) Natalia P. Rocha et al.   MEDIATORS OF INFLAMMATION

Validation of a prognostic index for Huntington's disease

(2016) Jeffrey D. Long et al.   MOVEMENT DISORDERS

The Prevalence of Huntington's Disease

(2016) Michael D. Rawlins et al.   NEUROEPIDEMIOLOGY

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

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

(2016) Jana Miniarikova et al.   Molecular Therapy-Nucleic Acids

Identification of Genetic Factors that Modify Clinical Onset of Huntington’s Disease

(2015) Jong-Min Lee et al.   CELL

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

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

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

Personalized gene silencing therapeutics for Huntington disease

(2014) C. Kay et al.   CLINICAL GENETICS

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

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

(2012) Richard Grondin et al.   BRAIN

Proteomic Analysis of Wild-type and Mutant Huntingtin-associated Proteins in Mouse Brains Identifies Unique Interactions and Involvement in Protein Synthesis

(2012) Brady P. Culver et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

CAG repeat expansion in Huntington disease determines age at onset in a fully dominant fashion

(2012) J.- M. Lee et al.   NEUROLOGY

“Huntingtin Holiday”: Progress toward an Antisense Therapy for Huntington's Disease

(2012) Xiao-Hong Lu et al.   NEURON

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

(2012) Holly B. Kordasiewicz et al.   NEURON

Indexing disease progression at study entry with individuals at-risk for Huntington disease

(2011) Ying Zhang et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART B-NEUROPSYCHIATRIC GENETICS

Widespread suppression of huntingtin with convection-enhanced delivery of siRNA

(2011) David K. Stiles et al.   EXPERIMENTAL NEUROLOGY

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

Proteolysis of Mutant Huntingtin Produces an Exon 1 Fragment That Accumulates as an Aggregated Protein in Neuronal Nuclei in Huntington Disease

(2010) Christian Landles et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Huntington's disease: from molecular pathogenesis to clinical treatment

(2010) Christopher A Ross et al.   LANCET NEUROLOGY

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

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