The prospects of CRISPR-based genome engineering in the treatment of neurodegenerative disorders

Haplotype-based stratification of Huntington's disease

(2017) Michael J Chao et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

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 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

(2017) Alex Mas Monteys et al.   MOLECULAR THERAPY

Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes

(2017) Brett T Staahl et al.   NATURE BIOTECHNOLOGY

CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs

(2017) Lixia Wang et al.   Protein & Cell

Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells

(2017) Xiaohong Xu et al.   Stem Cell Reports

Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders

(2016) Daria Wojtal et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Molecular Pathophysiology of Fragile X-Associated Tremor/Ataxia Syndrome and Perspectives for Drug Development

(2016) Teresa Botta-Orfila et al.   CEREBELLUM

A novel neurodevelopmental disorder associated with compound heterozygous variants in the huntingtin gene

(2016) Lance H Rodan et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9

(2016) John G Doench et al.   NATURE BIOTECHNOLOGY

Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells

(2016) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells

(2016) Daesik Kim et al.   NATURE BIOTECHNOLOGY

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

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

(2016) Ming Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Generation of an isogenic, gene-corrected iPSC line from a symptomatic 57-year-old female patient with frontotemporal dementia caused by a P301L mutation in the microtubule associated protein tau (MAPT) gene

(2016) Natakarn Nimsanor et al.   Stem Cell Research

Generation of an isogenic, gene-corrected iPSC line from a pre-symptomatic 28-year-old woman with an R406W mutation in the microtubule associated protein tau (MAPT) gene

(2016) Natakarn Nimsanor et al.   Stem Cell Research

Generation of an isogenic, gene-corrected iPSC line from a symptomatic 59-year-old female patient with frontotemporal dementia caused by an R406W mutation in the microtubule associated protein tau (MAPT) gene

(2016) Natakarn Nimsanor et al.   Stem Cell Research

Generation of a gene-corrected isogenic control hiPSC line derived from a familial Alzheimer's disease patient carrying a L150P mutation in presenilin 1

(2016) Anna Poon et al.   Stem Cell Research

Generation of a gene-corrected isogenic control cell line from an Alzheimer's disease patient iPSC line carrying a A79V mutation in PSEN1

(2016) Carlota Pires et al.   Stem Cell Research

Contracting CAG/CTG repeats using the CRISPR-Cas9 nickase

(2016) Cinzia Cinesi et al.   Nature Communications

Systematic quantification of HDR and NHEJ reveals effects of locus, nuclease, and cell type on genome-editing

(2016) Yuichiro Miyaoka et al.   Scientific Reports

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

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

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Modelling amyotrophic lateral sclerosis (ALS) using mutant and CAS9/CRISPR-corrected motor neurons from patients with C9ORF72 mutations reveals disease-specific cellular phenotypes

(2015) R. Mutihac et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Small molecule–triggered Cas9 protein with improved genome-editing specificity

(2015) Kevin M Davis et al.   Nature Chemical Biology

Rapid reverse genetic screening using CRISPR in zebrafish

(2015) Arish N Shah et al.   NATURE METHODS

Applications of CRISPR–Cas systems in neuroscience

(2015) Matthias Heidenreich et al.   NATURE REVIEWS NEUROSCIENCE

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Somatic CRISPR/Cas9-mediated tumour suppressor disruption enables versatile brain tumour modelling

(2015) Marc Zuckermann et al.   Nature Communications

Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons

(2015) Chul-Yong Park et al.   Cell Reports

Huntington disease

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

Repeat-mediated epigenetic dysregulation of the FMR1 gene in the fragile X-related disorders

(2015) Karen Usdin et al.   Frontiers in Genetics

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Conditional Knockouts Generated by Engineered CRISPR-Cas9 Endonuclease Reveal the Roles of Coronin in C. elegans Neural Development

(2014) Zhongfu Shen et al.   DEVELOPMENTAL CELL

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

(2014) Amber L Southwell et al.   MOLECULAR THERAPY

Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells

(2014) Xuebing Wu et al.   NATURE BIOTECHNOLOGY

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

CRISPR/Cas9-Mediated Gene Knock-Down in Post-Mitotic Neurons

(2014) Christoph Straub et al.   PLoS One

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Is Non-Homologous End-Joining Really an Inherently Error-Prone Process?

(2014) Mireille Bétermier et al.   PLoS Genetics

Repeat-mediated genetic and epigenetic changes at the FMR1 locus in the Fragile X-related disorders

(2014) Karen Usdin et al.   Frontiers in Genetics

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

Optical control of mammalian endogenous transcription and epigenetic states

(2013) Silvana Konermann et al.   NATURE

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

Seamless genome editing in human pluripotent stem cells using custom endonuclease–based gene targeting and the piggyBac transposon

(2013) Kosuke Yusa   Nature Protocols

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

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

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

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

(2012) J.- M. Lee et al.   NEUROLOGY

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

The chromosome 9 ALS and FTD locus is probably derived from a single founder

(2011) Kin Mok et al.   NEUROBIOLOGY OF AGING

Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS

(2011) Mariely DeJesus-Hernandez et al.   NEURON

A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

(2011) Alan E. Renton et al.   NEURON

The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA

(2010) Josiane E. Garneau et al.   NATURE

Prevalence and Instability of Fragile X Alleles

(2010) Amy Cronister et al.   OBSTETRICS AND GYNECOLOGY

CRISPR/Cas, the Immune System of Bacteria and Archaea

(2010) P. Horvath et al.   SCIENCE

C-terminal FUS/TLS mutations in familial and sporadic ALS in Germany

(2009) Carsten Drepper et al.   NEUROBIOLOGY OF AGING

Mutations in FUS, an RNA Processing Protein, Cause Familial Amyotrophic Lateral Sclerosis Type 6

(2009) C. Vance et al.   SCIENCE