Innovative Therapeutic Approaches for Huntington’s Disease: From Nucleic Acids to GPCR-Targeting Small Molecules

Discovery of the First Druggable GPR52 Antagonist to Treat Huntington’s Disease

(2021) Hidetoshi Komatsu   JOURNAL OF MEDICINAL CHEMISTRY

Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS

(2021) Tomoki Hirunagi et al.   Molecular Therapy-Nucleic Acids

Characterisation of inverse agonism of the orphan-G protein-coupled receptor GPR52 by cannabinoid ligands Cannabidiol and O-1918

(2021) Lisa A. Stott et al.   Heliyon

Structural basis of ligand recognition and self-activation of orphan GPR52

(2020) Xi Lin et al.   NATURE

Gene therapy for neurodegenerative disorders: advances, insights and prospects

(2020) Wei Chen et al.   Acta Pharmaceutica Sinica B

New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges

(2020) Sonia Messina et al.   Journal of Clinical Medicine

Discovery of Potent and Brain-Penetrant GPR52 Agonist that Suppresses Psychostimulant Behavior

(2020) Pingyuan Wang et al.   JOURNAL OF MEDICINAL CHEMISTRY

Investigational Assay for Haplotype Phasing of the Huntingtin Gene

(2020) Nenad Svrzikapa et al.   Molecular Therapy-Methods & Clinical Development

Cerebellar modulation of the reward circuitry and social behavior

(2019) Ilaria Carta et al.   SCIENCE

Targeting Huntingtin Expression in Patients with Huntington’s Disease

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

Emerging therapies in Huntington’s disease

(2019) Hassaan Bashir   Expert Review of Neurotherapeutics

Therapeutic Antisense Targeting of Huntingtin

(2019) Anne V. Smith et al.   DNA AND CELL BIOLOGY

Targeting Gpr52 lowers mutant HTT levels and rescues Huntington’s disease-associated phenotypes

(2018) Haikun Song et al.   BRAIN

Promoting the clearance of neurotoxic proteins in neurodegenerative disorders of ageing

(2018) Barry Boland et al.   NATURE REVIEWS DRUG DISCOVERY

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

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

Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease

(2018) Amber L. Southwell et al.   Science Translational Medicine

FTBMT, a Novel and Selective GPR52 Agonist, Demonstrates Antipsychotic-Like and Procognitive Effects in Rodents, Revealing a Potential Therapeutic Agent for Schizophrenia

(2017) Keiji Nishiyama et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Lipid Nanoparticle Systems for Enabling Gene Therapies

(2017) Pieter R. Cullis et al.   MOLECULAR THERAPY

Trends in GPCR drug discovery: new agents, targets and indications

(2017) Alexander S. Hauser et al.   NATURE REVIEWS DRUG DISCOVERY

Antisense oligonucleotides: the next frontier for treatment of neurological disorders

(2017) Carlo Rinaldi et al.   Nature Reviews Neurology

Mutant Huntingtin Disrupts the Nuclear Pore Complex

(2017) Jonathan C. Grima et al.   NEURON

Cellular selectivity of AAV serotypes for gene delivery in neurons and astrocytes by neonatal intracerebroventricular injection

(2017) Sean L. Hammond et al.   PLoS One

Juvenile Huntington’s Disease: Diagnostic and Treatment Considerations for the Psychiatrist

(2017) Joanna Quigley   Current Psychiatry Reports

Targeting CAG repeat RNAs reduces Huntington’s disease phenotype independently of huntingtin levels

(2016) Laura Rué et al.   JOURNAL OF CLINICAL INVESTIGATION

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

The Prevalence of Huntington's Disease

(2016) Michael D. Rawlins et al.   NEUROEPIDEMIOLOGY

The Biology of Huntingtin

(2016) Frédéric Saudou 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

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

Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia

(2015) Megan S. Keiser et al.   HUMAN MOLECULAR GENETICS

Novel Therapeutic GPCRs for Psychiatric Disorders

(2015) Hidetoshi Komatsu   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

A striatal-enriched intronic GPCR modulates huntingtin levels and toxicity

(2015) Yuwei Yao et al.   eLife

Huntington disease

(2015) Gillian P. Bates et al.   Nature Reviews Disease Primers

Mapping dopaminergic deficiencies in the substantia nigra/ventral tegmental area in schizophrenia

(2014) Matthew W. Rice et al.   Brain Structure & Function

Personalized gene silencing therapeutics for Huntington disease

(2014) C. Kay et al.   CLINICAL GENETICS

Discovery of the First Potent and Orally Available Agonist of the Orphan G-Protein-Coupled Receptor 52

(2014) Masaki Setoh et al.   JOURNAL OF MEDICINAL CHEMISTRY

Diagnostic criteria for Huntington's disease based on natural history

(2014) Ralf Reilmann et al.   MOVEMENT DISORDERS

Genetic modifiers of Huntington's disease

(2014) James F. Gusella et al.   MOVEMENT DISORDERS

Anatomical Transcriptome of G Protein-Coupled Receptors Leads to the Identification of a Novel Therapeutic Candidate GPR52 for Psychiatric Disorders

(2014) Hidetoshi Komatsu et al.   PLoS One

Choosing an animal model for the study of Huntington's disease

(2013) Mahmoud A. Pouladi et al.   NATURE REVIEWS NEUROSCIENCE

Analysis of Transduction Efficiency, Tropism and Axonal Transport of AAV Serotypes 1, 2, 5, 6, 8 and 9 in the Mouse Brain

(2013) Dominik F. Aschauer et al.   PLoS One

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

Induced Pluripotent Stem Cells from Patients with Huntington's Disease Show CAG-Repeat-Expansion-Associated Phenotypes

(2012) The HD iPSC Consortium   Cell Stem Cell

The incidence and prevalence of Huntington's disease: A systematic review and meta-analysis

(2012) Tamara Pringsheim et al.   MOVEMENT DISORDERS

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

(2012) Holly B. Kordasiewicz et al.   NEURON

Striatal neurons expressing full-length mutant huntingtin exhibit decreased N-cadherin and altered neuritogenesis

(2011) Surya A. Reis et al.   HUMAN MOLECULAR GENETICS

Development of biomarkers for Huntington's disease

(2011) David W Weir et al.   LANCET 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

Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease

(2010) Chiara Zuccato et al.   PHYSIOLOGICAL REVIEWS

A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference

(2009) Maria Stella Lombardi et al.   EXPERIMENTAL NEUROLOGY

Mutant Huntingtin N-terminal Fragments of Specific Size Mediate Aggregation and Toxicity in Neuronal Cells

(2009) Tamara Ratovitski et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

AAV9: a potential blood-brain barrier buster

(2009) Fredric P Manfredsson et al.   MOLECULAR THERAPY

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

Anatomical Profiling of G Protein-Coupled Receptor Expression

(2008) Jean B. Regard et al.   CELL

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

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