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

Striatal molecular alterations in HD gene carriers: a systematic review and meta-analysis of PET studies

(2017) Flavia Niccolini et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Locomotor speed control circuits in the caudal brainstem

(2017) Paolo Capelli et al.   NATURE

Developmental alterations in Huntington's disease neural cells and pharmacological rescue in cells and mice

(2017) Ryan G Lim et al.   NATURE NEUROSCIENCE

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

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

RNA toxicity induced by expanded CAG repeats in Huntington's disease

(2016) Eulàlia Martí   BRAIN PATHOLOGY

AAV viral vector delivery to the brain by shape-conforming MR-guided infusions

(2016) Krystof S. Bankiewicz et al.   JOURNAL OF CONTROLLED RELEASE

Identification of novel genetic causes of Rett syndrome-likephenotypes

(2016) Fátima Lopes et al.   JOURNAL OF MEDICAL GENETICS

Longitudinal Small-Animal PET Imaging of the zQ175 Mouse Model of Huntington Disease Shows In Vivo Changes of Molecular Targets in the Striatum and Cerebral Cortex

(2016) Jenny Häggkvist et al.   JOURNAL OF NUCLEAR MEDICINE

Loss of extra-striatal phosphodiesterase 10A expression in early premanifest Huntington's disease gene carriers

(2016) Heather Wilson et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice

(2016) Carmen Agustín-Pavón et al.   Molecular Neurodegeneration

Editing the epigenome: technologies for programmable transcription and epigenetic modulation

(2016) Pratiksha I Thakore et al.   NATURE METHODS

Postnatal and adult consequences of loss of huntingtin during development: Implications for Huntington's disease

(2016) Eduardo E. Arteaga-Bracho et al.   NEUROBIOLOGY OF DISEASE

Change in PDE10 across early Huntington disease assessed by [18F]MNI-659 and PET imaging

(2016) David S. Russell et al.   NEUROLOGY

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

Corticostriatal synaptic adaptations in Huntington's disease

(2015) Joshua L Plotkin et al.   CURRENT OPINION IN NEUROBIOLOGY

HD iPSC-derived neural progenitors accumulate in culture and are susceptible to BDNF withdrawal due to glutamate toxicity

(2015) Virginia B. Mattis et al.   HUMAN MOLECULAR GENETICS

Altered excitatory and inhibitory inputs to striatal medium-sized spiny neurons and cortical pyramidal neurons in the Q175 mouse model of Huntington's disease

(2015) Tim Indersmitten et al.   JOURNAL OF NEUROPHYSIOLOGY

RAN Translation in Huntington Disease

(2015) Monica Bañez-Coronel et al.   NEURON

Spatiotemporal regulation of Heterochromatin Protein 1- alpha oligomerization and dynamics in live cells

(2015) Elizabeth Hinde et al.   Scientific Reports

Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo

(2015) Alex Mas Monteys et al.   Molecular Therapy-Nucleic Acids

Huntington disease

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

Characterization of HTT Inclusion Size, Location, and Timing in the zQ175 Mouse Model of Huntington´s Disease: An In Vivo High-Content Imaging Study

(2015) Nikisha Carty et al.   PLoS One

Activating human genes with zinc finger proteins, transcription activator-like effectors and CRISPR/Cas9 for gene therapy and regenerative medicine

(2014) Charles A Gersbach et al.   EXPERT OPINION ON THERAPEUTIC TARGETS

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

(2014) Amber L Southwell et al.   MOLECULAR THERAPY

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

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

KRAB-Zinc Finger Proteins: A Repressor Family Displaying Multiple Biological Functions

(2013) Angelo Lupo et al.   CURRENT GENOMICS

Performance Evaluation of the Small-Animal nanoScan PET/MRI System

(2013) K. Nagy et al.   JOURNAL OF NUCLEAR MEDICINE

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

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

(2012) Richard Grondin et al.   BRAIN

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

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

(2012) Holly B. Kordasiewicz et al.   NEURON

Comprehensive Behavioral and Molecular Characterization of a New Knock-In Mouse Model of Huntington’s Disease: zQ175

(2012) Liliana B. Menalled et al.   PLoS One

Characterization of Neurophysiological and Behavioral Changes, MRI Brain Volumetry and 1H MRS in zQ175 Knock-In Mouse Model of Huntington's Disease

(2012) Taneli Heikkinen et al.   PLoS One

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

Widespread suppression of huntingtin with convection-enhanced delivery of siRNA

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

KAP1 Protein: An Enigmatic Master Regulator of the Genome

(2011) Sushma Iyengar et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

National Electrical Manufacturers Association NU-4 Performance Evaluation of the PET Component of the NanoPET/CT Preclinical PET/CT Scanner

(2011) I. Szanda et al.   JOURNAL OF NUCLEAR MEDICINE

Chronic imaging of cortical sensory map dynamics using a genetically encoded calcium indicator

(2011) Matthias Minderer et al.   JOURNAL OF PHYSIOLOGY-LONDON

Microarray Analysis of Gene Expression Profiles in Cells Transfected With Nonviral Vectors

(2011) Sarah A Plautz 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

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

(2010) Keith T. Gagnon et al.   BIOCHEMISTRY

Huntington's disease: from molecular pathogenesis to clinical treatment

(2010) Christopher A Ross et al.   LANCET NEUROLOGY

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease

(2010) Chiara Zuccato et al.   PHYSIOLOGICAL REVIEWS

Nucleosome-mediated cooperativity between transcription factors

(2010) L. A. Mirny   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Derivation of Huntington's Disease-Affected Human Embryonic Stem Cell Lines

(2010) Cara K. Bradley et al.   STEM CELLS AND DEVELOPMENT

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

(2009) Edith L. Pfister et al.   CURRENT 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

Systematic behavioral evaluation of Huntington's disease transgenic and knock-in mouse models

(2009) Liliana Menalled et al.   NEUROBIOLOGY OF DISEASE

Mutation Detection by Real-Time PCR: A Simple, Robust and Highly Selective Method

(2009) John Morlan et al.   PLoS One

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE