Creating cell and animal models of human disease by genome editing using CRISPR/Cas9

SaCas9 Requires 5′-NNGRRT-3′ PAM for Sufficient Cleavage and Possesses Higher Cleavage Activity than SpCas9 or FnCpf1 in Human Cells

(2018) Haihua Xie et al.   Biotechnology Journal

A Huntingtin Knockin Pig Model Recapitulates Features of Selective Neurodegeneration in Huntington’s Disease

(2018) Sen Yan et al.   CELL

Targeted gene disruption by use of CRISPR/Cas9 ribonucleoprotein complexes in the water flea Daphnia pulex

(2018) Chizue Hiruta et al.   GENES TO CELLS

Optimized RNP transfection for highly efficient CRISPR/Cas9-mediated gene knockout in primary T cells

(2018) Akiko Seki et al.   JOURNAL OF EXPERIMENTAL MEDICINE

An optimized electroporation approach for efficient CRISPR/Cas9 genome editing in murine zygotes

(2018) Simon E. Tröder et al.   PLoS One

Strategies for In Vivo Genome Editing in Nondividing Cells

(2018) Fatemeharefeh Nami et al.   TRENDS IN BIOTECHNOLOGY

TALEN-Induced Double-Strand Break Repair of CTG Trinucleotide Repeats

(2018) Valentine Mosbach et al.   Cell Reports

SaCas9 Requires 5′-NNGRRT-3′ PAM for Sufficient Cleavage and Possesses Higher Cleavage Activity than SpCas9 or FnCpf1 in Human Cells

(2018) Haihua Xie et al.   Biotechnology Journal

Use of gene-editing technology to introduce targeted modifications in pigs

(2018) Junghyun Ryu et al.   Journal of Animal Science and Biotechnology

Generation of Hutat2:Fc Knockin Primary Human Monocytes Using CRISPR/Cas9

(2018) Bowen Wang et al.   Molecular Therapy-Nucleic Acids

CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer’s Disease

(2018) Bence György et al.   Molecular Therapy-Nucleic Acids

Gene editing in the context of an increasingly complex genome

(2018) K. Blighe et al.   BMC GENOMICS

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

(2018) Johnny H. Hu et al.   NATURE

Highly efficient genome editing by CRISPR-Cpf1 using CRISPR RNA with a uridinylate-rich 3′-overhang

(2018) Su Bin Moon et al.   Nature Communications

CRISPR-Cpf1-mediated genome editing and gene regulation in human cells

(2018) Tianwen Li et al.   BIOTECHNOLOGY ADVANCES

Effective CRISPR/Cas9-based nucleotide editing in zebrafish to model human genetic cardiovascular disorders

(2018) Federico Tessadori et al.   Disease Models & Mechanisms

From “the Worm” to “the Worms” and Back Again: The Evolutionary Developmental Biology of Nematodes

(2018) Eric S. Haag et al.   GENETICS

Tissue-Specific Gene Inactivation inXenopus laevis: Knockout oflhx1in the Kidney with CRISPR/Cas9

(2017) Bridget D. DeLay et al.   GENETICS

Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications

(2017) Chang Liu et al.   JOURNAL OF CONTROLLED RELEASE

Developmental history and application of CRISPR in human disease

(2017) Puping Liang et al.   JOURNAL OF GENE MEDICINE

In vivo genome editing via the HITI method as a tool for gene therapy

(2017) Keiichiro Suzuki et al.   JOURNAL OF HUMAN GENETICS

Simplified CRISPR tools for efficient genome editing and streamlined protocols for their delivery into mammalian cells and mouse zygotes

(2017) Ashley M. Jacobi et al.   METHODS

Correction of a pathogenic gene mutation in human embryos

(2017) Hong Ma et al.   NATURE

Concise Review: Induced Pluripotent Stem Cell Research in the Era of Precision Medicine

(2017) Takashi Hamazaki et al.   STEM CELLS

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

(2017) Eunji Kim et al.   Nature Communications

Tissue-Specific Gene Inactivation inXenopus laevis: Knockout oflhx1in the Kidney with CRISPR/Cas9

(2017) Bridget D. DeLay et al.   GENETICS

Highly efficient gene inactivation by adenoviral CRISPR/Cas9 in human primary cells

(2017) Olaf Voets et al.   PLoS One

CRISPR/Cas9-mediated gene manipulation to create single-amino-acid-substituted and floxed mice with a cloning-free method

(2017) Xiaolong Ma et al.   Scientific Reports

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

(2016) Jason B. Miller et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

CRISPR/Cas9 Immune System as a Tool for Genome Engineering

(2016) Magdalena Hryhorowicz et al.   ARCHIVUM IMMUNOLOGIAE ET THERAPIAE EXPERIMENTALIS

Novel Thrombotic Function of a Human SNP in STXBP5 Revealed by CRISPR/Cas9 Gene Editing in MiceHighlights

(2016) Qiuyu Martin Zhu et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Insert, remove or replace: A highly advanced genome editing system using CRISPR/Cas9

(2016) S. Antony Ceasar et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Electroporation of Cas9 protein/sgRNA into early pronuclear zygotes generates non-mosaic mutants in the mouse

(2016) Masakazu Hashimoto et al.   DEVELOPMENTAL BIOLOGY

Using CRISPR/Cas to study gene function and model diseasein vivo

(2016) Darjus F. Tschaharganeh et al.   FEBS Journal

CRISPRs for Optimal Targeting: Delivery of CRISPR Components as DNA, RNA, and Protein into Cultured Cells and Single-Cell Embryos

(2016) Evguenia Kouranova et al.   HUMAN GENE THERAPY

Methods for Optimizing CRISPR-Cas9 Genome Editing Specificity

(2016) Josh Tycko et al.   MOLECULAR CELL

Current Progress in Therapeutic Gene Editing for Monogenic Diseases

(2016) Versha Prakash et al.   MOLECULAR THERAPY

In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

(2016) Benjamin Bakondi et al.   MOLECULAR THERAPY

Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9

(2016) Dominik Paquet et al.   NATURE

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

Parkinson-associated risk variant in distal enhancer of α-synuclein modulates target gene expression

(2016) Frank Soldner et al.   NATURE

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Patterns of CRISPR/Cas9 activity in plants, animals and microbes

(2016) Luisa Bortesi et al.   PLANT BIOTECHNOLOGY JOURNAL

Advances in therapeutic CRISPR/Cas9 genome editing

(2016) Nataša Savić et al.   Translational Research

Highly efficient generation of GGTA1 knockout pigs using a combination of TALEN mRNA and magnetic beads with somatic cell nuclear transfer

(2016) Chong FENG et al.   Journal of Integrative Agriculture

CRISPR-Cas9D10A nickase-based genotypic and phenotypic screening to enhance genome editing

(2016) Ting-Wei Will Chiang et al.   Scientific Reports

Application of CRISPR/Cas9 genome editing to the study and treatment of disease

(2015) Andrea Pellagatti et al.   ARCHIVES OF TOXICOLOGY

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

(2015) Bernd Zetsche et al.   CELL

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers

(2015) Isaac B Hilton et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

An updated evolutionary classification of CRISPR–Cas systems

(2015) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

Large animal models of rare genetic disorders: sheep as phenotypically relevant models of human genetic disease

(2015) Ashish R. Pinnapureddy et al.   Orphanet Journal of Rare Diseases

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

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

(2015) Marc Zuckermann et al.   Nature Communications

Derivation of Ethnically Diverse Human Induced Pluripotent Stem Cell Lines

(2015) Eun Ah Chang et al.   Scientific Reports

Efficient generation of gene-modified pigs via injection of zygote with Cas9/sgRNA

(2015) Yong Wang et al.   Scientific Reports

Targeted Germline Modifications in Rats Using CRISPR/Cas9 and Spermatogonial Stem Cells

(2015) Karen M. Chapman et al.   Cell Reports

Vascular Smooth Muscle Cells From Hypertensive Patient-Derived Induced Pluripotent Stem Cells to Advance Hypertension Pharmacogenomics

(2015) Nikolett M. Biel et al.   Stem Cells Translational Medicine

CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation

(2015) X. Han et al.   Science Advances

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA

(2014) Hiroshi Nishimasu et al.   CELL

Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos

(2014) Yuyu Niu et al.   CELL

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Targeted Genome Editing Tools for Disease Modeling and Gene Therapy

(2014) Mi Cai et al.   CURRENT GENE THERAPY

TALENs Mediate Efficient and Heritable Mutation of Endogenous Genes in the Marine Annelid Platynereis dumerilii

(2014) Stephanie Bannister et al.   GENETICS

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

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

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Broad specificity profiling of TALENs results in engineered nucleases with improved DNA-cleavage specificity

(2014) John P Guilinger et al.   NATURE METHODS

Generating genetically modified mice using CRISPR/Cas-mediated genome engineering

(2014) Hui Yang et al.   Nature Protocols

Investigating human disease using stem cell models

(2014) Jared L. Sterneckert et al.   NATURE REVIEWS GENETICS

Rapid generation of mouse models with defined point mutations by the CRISPR/Cas9 system

(2014) Masafumi Inui et al.   Scientific Reports

Generation of muscular dystrophy model rats with a CRISPR/Cas system

(2014) Katsuyuki Nakamura et al.   Scientific Reports

Genetic heterogeneity in breast cancer: the road to personalized medicine?

(2013) Richard D Baird et al.   BMC Medicine

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression

(2013) Lei S. Qi et al.   CELL

CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes

(2013) Luke A. Gilbert et al.   CELL

Efficient genome editing in plants using a CRISPR/Cas system

(2013) Zhengyan Feng et al.   CELL RESEARCH

Enhanced Efficiency of Human Pluripotent Stem Cell Genome Editing through Replacing TALENs with CRISPRs

(2013) Qiurong Ding et al.   Cell Stem Cell

Highly efficient CRISPR/Cas9-mediated knock-in in zebrafish by homology-independent DNA repair

(2013) T. O. Auer et al.   GENOME RESEARCH

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

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

Simultaneous generation and germline transmission of multiple gene mutations in rat using CRISPR-Cas systems

(2013) Wei Li et al.   NATURE BIOTECHNOLOGY

Heritable and Precise Zebrafish Genome Editing Using a CRISPR-Cas System

(2013) Woong Y. Hwang et al.   PLoS One

Efficient multiplex biallelic zebrafish genome editing using a CRISPR nuclease system

(2013) Li-En Jao et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis

(2013) Z. Hou et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Genomic responses in mouse models poorly mimic human inflammatory diseases

(2013) Junhee Seok et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Protospacer recognition motifs

(2013) Shiraz A. Shah et al.   RNA Biology

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

Genome engineering of mammalian haploid embryonic stem cells using the Cas9/RNA system

(2013) Takuro Horii et al.   PeerJ

A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models

(2012) Qiurong Ding et al.   Cell Stem Cell

FLASH assembly of TALENs for high-throughput genome editing

(2012) Deepak Reyon et al.   NATURE BIOTECHNOLOGY

Diagnosing the decline in pharmaceutical R&D efficiency

(2012) Jack W. Scannell et al.   NATURE REVIEWS DRUG DISCOVERY

Targeted transcriptional activation of silent oct4 pluripotency gene by combining designer TALEs and inhibition of epigenetic modifiers

(2012) Sebastian Bultmann et al.   NUCLEIC ACIDS RESEARCH

Structural Basis for Sequence-Specific Recognition of DNA by TAL Effectors

(2012) D. Deng et al.   SCIENCE

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

(2012) M. Jinek et al.   SCIENCE

Site-specific integration and tailoring of cassette design for sustainable gene transfer

(2011) Angelo Lombardo et al.   NATURE METHODS

XanthomonasAvrBs3 Family-Type III Effectors: Discovery and Function

(2010) Jens Boch et al.   Annual Review of Phytopathology

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

CRISPR interference: RNA-directed adaptive immunity in bacteria and archaea

(2010) Luciano A. Marraffini et al.   NATURE REVIEWS GENETICS

Directed evolution of homing endonuclease I-SceI with altered sequence specificity

(2009) Z. Chen et al.   PROTEIN ENGINEERING DESIGN & SELECTION

Rapid “Open-Source” Engineering of Customized Zinc-Finger Nucleases for Highly Efficient Gene Modification

(2008) Morgan L. Maeder et al.   MOLECULAR CELL