Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases

CRISPR-Cas9 Mediated Gene-Silencing of the Mutant Huntingtin Gene in an In Vitro Model of Huntington’s Disease

(2017) Nivya Kolli et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

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

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

Therapies targeting DNA and RNA in Huntington's disease

(2017) Edward J Wild et al.   LANCET NEUROLOGY

CRISPR/Cas9-Induced (CTG⋅CAG) n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing

(2017) Ellen L. van Agtmaal et al.   MOLECULAR THERAPY

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

(2017) Alex Mas Monteys et al.   MOLECULAR THERAPY

Polyglutamine spinocerebellar ataxias — from genes to potential treatments

(2017) Henry L. Paulson et al.   NATURE REVIEWS NEUROSCIENCE

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

(2017) Nicolas Merienne et al.   Cell Reports

Alterations in mRNA 3′ UTR Isoform Abundance Accompany Gene Expression Changes in Human Huntington’s Disease Brains

(2017) Lindsay Romo et al.   Cell Reports

Leveraging Rules of Nonsense-Mediated mRNA Decay for Genome Engineering and Personalized Medicine

(2016) Maximilian W. Popp et al.   CELL

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9

(2016) Jun Wan Shin et al.   HUMAN MOLECULAR GENETICS

Discovery of Therapeutic Approaches for Polyglutamine Diseases: A Summary of Recent Efforts

(2016) Sofia Esteves et al.   MEDICINAL RESEARCH REVIEWS

Identifying and Visualizing Functional PAM Diversity across CRISPR-Cas Systems

(2016) Ryan T. Leenay et al.   MOLECULAR CELL

The Biology of Huntingtin

(2016) Frédéric Saudou et al.   NEURON

Precision Modulation of Neurodegenerative Disease-Related Gene Expression in Human iPSC-Derived Neurons

(2016) Sabrina Mahalia Heman-Ackah et al.   Scientific Reports

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

Huntington disease

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

Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects

(2014) Bin Shen et al.   NATURE METHODS

Highly Specific Contractions of a Single CAG/CTG Trinucleotide Repeat by TALEN in Yeast

(2014) Guy-Franck Richard et al.   PLoS One

Comparison of non-canonical PAMs for CRISPR/Cas9-mediated DNA cleavage in human cells

(2014) Yilan Zhang et al.   Scientific Reports

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases

(2013) S. W. Cho et al.   GENOME RESEARCH

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression

(2013) Agnieszka Fiszer et al.   NUCLEIC ACIDS RESEARCH

An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases

(2012) Agnieszka Fiszer et al.   BMC MOLECULAR BIOLOGY

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

(2012) Dongbo Yu et al.   CELL

Genetic Correction of Huntington's Disease Phenotypes in Induced Pluripotent Stem Cells

(2012) Mahru C. An et al.   Cell Stem Cell

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

(2012) Holly B. Kordasiewicz et al.   NEURON

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

Control of human  -globin mRNA stability and its impact on beta-thalassemia phenotype

(2011) I. Peixeiro et al.   HAEMATOLOGICA

Mechanisms and Consequences of Alternative Polyadenylation

(2011) Dafne Campigli Di Giammartino et al.   MOLECULAR CELL

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

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