Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts

Delivery of episomal vectors into primary cells by means of commercial transfection reagents

(2015) Na Rae Han et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Ubiquitin: a potential cerebrospinal fluid progression marker in Huntington's disease

(2015) T. Vinther-Jensen et al.   EUROPEAN JOURNAL OF NEUROLOGY

Making (anti-) sense out of huntingtin levels in Huntington disease

(2015) Melvin M Evers et al.   Molecular Neurodegeneration

Multivariate clustering of progression profiles reveals different depression patterns in prodromal Huntington disease.

(2015) Ji-in Kim et al.   NEUROPSYCHOLOGY

Shortening trinucleotide repeats using highly specific endonucleases: a possible approach to gene therapy?

(2015) Guy-Franck Richard   TRENDS IN GENETICS

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

Mutant Huntingtin Affects Cortical Progenitor Cell Division and Development of the Mouse Neocortex

(2014) M. Molina-Calavita et al.   JOURNAL OF NEUROSCIENCE

Huntingtin Is Required for Normal Excitatory Synapse Development in Cortical and Striatal Circuits

(2014) S. U. McKinstry et al.   JOURNAL OF NEUROSCIENCE

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

Huntington disease: natural history, biomarkers and prospects for therapeutics

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

Direct activation of human and mouse Oct4 genes using engineered TALE and Cas9 transcription factors

(2014) Jiabiao Hu et al.   NUCLEIC ACIDS RESEARCH

Trehalose Reverses Cell Malfunction in Fibroblasts from Normal and Huntington's Disease Patients Caused by Proteosome Inhibition

(2014) Maria Angeles Fernandez-Estevez et al.   PLoS One

Potential function for the Huntingtin protein as a scaffold for selective autophagy

(2014) Joseph Ochaba et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A multicolor panel of TALE-KRAB based transcriptional repressor vectors enabling knockdown of multiple gene targets

(2014) Zhonghui Zhang et al.   Scientific Reports

Clinical and Biomarker Changes in Premanifest Huntington Disease Show Trial Feasibility: A Decade of the PREDICT-HD Study

(2014) Jane S. Paulsen et al.   Frontiers in Aging Neuroscience

Dysfunction of the CNS-Heart Axis in Mouse Models of Huntington's Disease

(2014) Michal Mielcarek et al.   PLoS Genetics

Kruppel-associated box (KRAB) proteins in the adaptive immune system

(2014) Francesca R Santoni de Sio   Nucleus

Skeletal muscle pathology in Huntington's disease

(2014) Daniel Zielonka et al.   Frontiers in Physiology

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

(2012) Holly B. Kordasiewicz et al.   NEURON

Highly efficient generation of heritable zebrafish gene mutations using homo- and heterodimeric TALENs

(2012) Lindsay Cade et al.   NUCLEIC ACIDS RESEARCH

Temporal Separation of Aggregation and Ubiquitination during Early Inclusion Formation in Transgenic Mice Carrying the Huntington’s Disease Mutation

(2012) Belvin Gong 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

Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington's disease affected neuronal cells for reduction of huntingtin

(2011) Scott D. Olson et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

(2011) Tomas Cermak et al.   NUCLEIC ACIDS RESEARCH

De novo-engineered transcription activator-like effector (TALE) hybrid nuclease with novel DNA binding specificity creates double-strand breaks

(2011) M. M. Mahfouz et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Mitochondria and Huntington's Disease Pathogenesis

(2010) Susan E. Browne   Annals of the New York Academy of Sciences

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures

(2010) Yannick Doyon et al.   NATURE METHODS

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

The relationship between impairment of voluntary movements and cognitive impairment in Huntington’s disease

(2009) Jiří Klempíř et al.   JOURNAL OF NEUROLOGY

Excitotoxic Neuronal Death and the Pathogenesis of Huntington's Disease

(2008) Ana María Estrada Sánchez et al.   ARCHIVES OF MEDICAL RESEARCH