CRISPR editing in biological and biomedical investigation

Biallelic insertion of a transcriptional terminator via the CRISPR/Cas9 system efficiently silences expression of protein-coding and non-coding RNA genes

(2017) Yangyang Liu et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

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

Transgenic Clustered Regularly Interspaced Short Palindromic Repeat/Cas9-Mediated Viral Gene Targeting for Antiviral Therapy of Bombyx mori Nucleopolyhedrovirus

(2017) Shuqing Chen et al.   JOURNAL OF VIROLOGY

Editing the genome of hiPSC with CRISPR/Cas9: disease models

(2017) Andrew R. Bassett   MAMMALIAN GENOME

High‐throughput CRISPRi phenotyping identifies new essential genes in Streptococcus pneumoniae

(2017) Xue Liu et al.   Molecular Systems Biology

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

(2017) Alex Mas Monteys et al.   MOLECULAR THERAPY

Precise base editing in rice, wheat and maize with a Cas9-cytidine deaminase fusion

(2017) Yuan Zong et al.   NATURE BIOTECHNOLOGY

CRISPR–Cas9 epigenome editing enables high-throughput screening for functional regulatory elements in the human genome

(2017) Tyler S Klann et al.   NATURE BIOTECHNOLOGY

Targeted base editing in rice and tomato using a CRISPR-Cas9 cytidine deaminase fusion

(2017) Zenpei Shimatani et al.   NATURE BIOTECHNOLOGY

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

(2017) Y Bill Kim et al.   NATURE BIOTECHNOLOGY

Highly efficient RNA-guided base editing in mouse embryos

(2017) Kyoungmi Kim et al.   NATURE BIOTECHNOLOGY

Genome-wide target specificities of CRISPR RNA-guided programmable deaminases

(2017) Daesik Kim et al.   NATURE BIOTECHNOLOGY

CRISPR-STOP: gene silencing through base-editing-induced nonsense mutations

(2017) Cem Kuscu et al.   NATURE METHODS

Therapeutic genome engineering via CRISPR-Cas systems

(2017) Ana M. Moreno et al.   Wiley Interdisciplinary Reviews-Systems Biology and Medicine

Stabilization of Foxp3 expression by CRISPR-dCas9-based epigenome editing in mouse primary T cells

(2017) Masahiro Okada et al.   Epigenetics & Chromatin

Locus-specific histone deacetylation using a synthetic CRISPR-Cas9-based HDAC

(2017) Deborah Y. Kwon et al.   Nature Communications

Seed-effect modeling improves the consistency of genome-wide loss-of-function screens and identifies synthetic lethal vulnerabilities in cancer cells

(2017) Alok Jaiswal et al.   Genome Medicine

Precision engineering for PRRSV resistance in pigs: Macrophages from genome edited pigs lacking CD163 SRCR5 domain are fully resistant to both PRRSV genotypes while maintaining biological function

(2017) Christine Burkard et al.   PLoS Pathogens

Site-specific recruitment of epigenetic factors with a modular CRISPR/Cas system

(2017) Tobias Anton et al.   Nucleus

Site-specific recruitment of epigenetic factors with a modular CRISPR/Cas system

(2017) Tobias Anton et al.   Nucleus

Structure and Engineering of Francisella novicida Cas9

(2016) Hisato Hirano et al.   CELL

Editing DNA Methylation in the Mammalian Genome

(2016) X. Shawn Liu et al.   CELL

Genome editing revolutionize the creation of genetically modified pigs for modeling human diseases

(2016) Jing Yao et al.   HUMAN GENETICS

Production of α1,3-galactosyltransferase targeted pigs using transcription activator-like effector nuclease-mediated genome editing technology

(2016) Jung-Taek Kang et al.   Journal of Veterinary Science

Replacement of Porcine CD163 Scavenger Receptor Cysteine-Rich Domain 5 with a CD163-Like Homolog Confers Resistance of Pigs to Genotype 1 but Not Genotype 2 Porcine Reproductive and Respiratory Syndrome Virus

(2016) Kevin D. Wells et al.   JOURNAL OF VIROLOGY

CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy

(2016) Thierry VandenDriessche et al.   MOLECULAR THERAPY

Autism-like behaviours and germline transmission in transgenic monkeys overexpressing MeCP2

(2016) Zhen Liu et al.   NATURE

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

(2016) Keiichiro Suzuki et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Targeted DNA demethylation in vivo using dCas9–peptide repeat and scFv–TET1 catalytic domain fusions

(2016) Sumiyo Morita et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Editing the epigenome: technologies for programmable transcription and epigenetic modulation

(2016) Pratiksha I Thakore et al.   NATURE METHODS

Efficient, footprint-free human iPSC genome editing by consolidation of Cas9/CRISPR and piggyBac technologies

(2016) Gang Wang et al.   Nature Protocols

In vivo genome editing — growing in strength

(2016) Linda Koch   NATURE REVIEWS GENETICS

Repurposing the CRISPR-Cas9 system for targeted DNA methylation

(2016) Aleksandar Vojta et al.   NUCLEIC ACIDS RESEARCH

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

Structures of a CRISPR-Cas9 R-loop complex primed for DNA cleavage

(2016) F. Jiang et al.   SCIENCE

C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector

(2016) Omar O. Abudayyeh et al.   SCIENCE

Efficient production of biallelicGGTA1knockout pigsbycytoplasmic microinjection of CRISPR/Cas9 into zygotes

(2016) Bjoern Petersen et al.   XENOTRANSPLANTATION

Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

(2016) Francis P. Pankowicz et al.   Nature Communications

RS-1 enhances CRISPR/Cas9- and TALEN-mediated knock-in efficiency

(2016) Jun Song et al.   Nature Communications

One-step generation of triple gene-targeted pigs using CRISPR/Cas9 system

(2016) Xianlong Wang et al.   Scientific Reports

Reprogrammable CRISPR/Cas9-based system for inducing site-specific DNA methylation

(2016) James I. McDonald et al.   Biology Open

A Cas9 Variant for Efficient Generation of Indel-Free Knockin or Gene-Corrected Human Pluripotent Stem Cells

(2016) Sara E. Howden et al.   Stem Cell Reports

A CRISPR-based approach for targeted DNA demethylation

(2016) Xingxing Xu et al.   Cell Discovery

Efficient production of multi-modified pigs for xenotransplantation by ‘combineering’, gene stacking and gene editing

(2016) Konrad Fischer et al.   Scientific Reports

CRISPR-Cas targeted plasmid integration into mammalian cells via non-homologous end joining

(2015) Ravichandra Bachu et al.   BIOTECHNOLOGY AND BIOENGINEERING

Genome-wide CRISPR Screen in a Mouse Model of Tumor Growth and Metastasis

(2015) Sidi Chen et al.   CELL

A Genome-wide CRISPR Screen in Primary Immune Cells to Dissect Regulatory Networks

(2015) Oren Parnas et al.   CELL

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

(2015) Bernd Zetsche et al.   CELL

Inhibition of nonhomologous end joining to increase the specificity of CRISPR/Cas9 genome editing

(2015) Supriya V. Vartak et al.   FEBS Journal

Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9

(2015) Yongchang Chen et al.   HUMAN MOLECULAR GENETICS

Conrad Waddington and the origin of epigenetics

(2015) D. Noble   JOURNAL OF EXPERIMENTAL BIOLOGY

The role of genetically engineered pigs in xenotransplantation research

(2015) David KC Cooper et al.   JOURNAL OF PATHOLOGY

Generation of α1,3-galactosyltransferase and cytidine monophospho-N-acetylneuraminic acid hydroxylase gene double-knockout pigs

(2015) Shuji MIYAGAWA et al.   JOURNAL OF REPRODUCTION AND DEVELOPMENT

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

(2015) Benjamin P. Kleinstiver et al.   NATURE

Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex

(2015) Silvana Konermann et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

(2015) Takeshi Maruyama et al.   NATURE BIOTECHNOLOGY

Gene-edited pigs are protected from porcine reproductive and respiratory syndrome virus

(2015) Kristin M Whitworth et al.   NATURE BIOTECHNOLOGY

Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition

(2015) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

Discovery of cancer drug targets by CRISPR-Cas9 screening of protein domains

(2015) Junwei Shi et al.   NATURE BIOTECHNOLOGY

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

(2015) Isaac B Hilton et al.   NATURE BIOTECHNOLOGY

Adaptation and possible ancient interspecies introgression in pigs identified by whole-genome sequencing

(2015) Huashui Ai et al.   NATURE GENETICS

Functional annotation of native enhancers with a Cas9–histone demethylase fusion

(2015) Nicola A Kearns et al.   NATURE METHODS

Chromatin regulation at the frontier of synthetic biology

(2015) Albert J. Keung et al.   NATURE REVIEWS GENETICS

High-throughput functional genomics using CRISPR–Cas9

(2015) Ophir Shalem et al.   NATURE REVIEWS GENETICS

Cas9-mediated targeting of viral RNA in eukaryotic cells

(2015) Aryn A. Price et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Making the cut

(2015) J. Travis   SCIENCE

Genome-wide inactivation of porcine endogenous retroviruses (PERVs)

(2015) L. Yang et al.   SCIENCE

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

(2015) C. Long 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

Modeling Disease In Vivo With CRISPR/Cas9

(2015) Lukas E. Dow   TRENDS IN MOLECULAR MEDICINE

Use of the CRISPR/Cas9 system as an intracellular defense against HIV-1 infection in human cells

(2015) Hsin-Kai Liao et al.   Nature Communications

CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus

(2015) Vyas Ramanan et al.   Scientific Reports

Pharmacological inhibition of DNA-PK stimulates Cas9-mediated genome editing

(2015) Francis Robert et al.   Genome Medicine

CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

(2015) Puping Liang et al.   Protein & Cell

Huntington disease

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

The basic building blocks and evolution of CRISPR–Cas systems

(2015) Kira S. Makarova et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Analysis of Irradiation on the Clinical Effectiveness of Allogenic Tissue When Used for Primary Anterior Cruciate Ligament Reconstruction

(2014) Sam Si-Hyeong Park et al.   AMERICAN JOURNAL OF SPORTS MEDICINE

Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation

(2014) Luke A. Gilbert 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

One-step generation of p53 gene biallelic mutant Cynomolgus monkey via the CRISPR/Cas system

(2014) Haifeng Wan et al.   CELL RESEARCH

TALEN-Mediated Gene Mutagenesis in Rhesus and Cynomolgus Monkeys

(2014) Hailiang Liu et al.   Cell Stem Cell

DNA Double-strand Break Repair in a Cellular Context

(2014) A. Shibata et al.   CLINICAL ONCOLOGY

The progress and future of enhancing antiviral capacity by transgenic technology in the silkworm Bombyx mori

(2014) Liang Jiang et al.   INSECT BIOCHEMISTRY AND MOLECULAR BIOLOGY

CRISPR-Cas Systems: Prokaryotes Upgrade to Adaptive Immunity

(2014) Rodolphe Barrangou et al.   MOLECULAR CELL

Bubble Liposomes and Ultrasound Exposure Improve Localized Morpholino Oligomer Delivery into the Skeletal Muscles of Dystrophic mdx Mice

(2014) Yoichi Negishi et al.   MOLECULAR PHARMACEUTICS

High-throughput screening of a CRISPR/Cas9 library for functional genomics in human cells

(2014) Yuexin Zhou et al.   NATURE

Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification

(2014) John P Guilinger et al.   NATURE BIOTECHNOLOGY

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Improved vectors and genome-wide libraries for CRISPR screening

(2014) Neville E Sanjana et al.   NATURE METHODS

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Allele-Specific Silencing of Mutant Huntingtin in Rodent Brain and Human Stem Cells

(2014) Valérie Drouet et al.   PLoS One

CCR5 Gene Disruption via Lentiviral Vectors Expressing Cas9 and Single Guided RNA Renders Cells Resistant to HIV-1 Infection

(2014) Weiming Wang et al.   PLoS One

RNA-guided endonuclease provides a therapeutic strategy to cure latent herpesviridae infection

(2014) J. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection

(2014) Lin Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection

(2014) W. Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Generation of GGTA1 biallelic knockout pigs via zinc-finger nucleases and somatic cell nuclear transfer

(2014) Lei Bao et al.   Science China-Life Sciences

Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9

(2014) Shota Nakade et al.   Nature Communications

Homologous recombination in human embryonic stem cells using CRISPR/Cas9 nickase and a long DNA donor template

(2014) Zhili Rong et al.   Protein & Cell

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

(2014) Steven Lin et al.   eLife

The CRISPR/Cas9 System Facilitates Clearance of the Intrahepatic HBV Templates In Vivo

(2014) Su-Ru Lin et al.   Molecular Therapy-Nucleic Acids

Targeting Hepatitis B Virus With CRISPR/Cas9

(2014) Christoph Seeger et al.   Molecular Therapy-Nucleic Acids

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

(2013) Lei S. Qi et al.   CELL

Dynamic Imaging of Genomic Loci in Living Human Cells by an Optimized CRISPR/Cas System

(2013) Baohui Chen et al.   CELL

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

(2013) F. Ann Ran et al.   CELL

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

(2013) Luke A. Gilbert et al.   CELL

Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library

(2013) Hiroko Koike-Yusa et al.   NATURE BIOTECHNOLOGY

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

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

RNA-guided gene activation by CRISPR-Cas9–based transcription factors

(2013) Pablo Perez-Pinera et al.   NATURE METHODS

CRISPR RNA–guided activation of endogenous human genes

(2013) Morgan L Maeder et al.   NATURE METHODS

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

(2013) Rebecca J. Fairclough et al.   NATURE REVIEWS GENETICS

Programmable repression and activation of bacterial gene expression using an engineered CRISPR-Cas system

(2013) David Bikard et al.   NUCLEIC ACIDS RESEARCH

Optimization of scarless human stem cell genome editing

(2013) Luhan Yang et al.   NUCLEIC ACIDS RESEARCH

Mammalian Base Excision Repair: the Forgotten Archangel

(2013) Grigory L. Dianov et al.   NUCLEIC ACIDS RESEARCH

Blastocyst complementation generates exogenic pancreas in vivo in apancreatic cloned pigs

(2013) H. Matsunari 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

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Genetic Screens in Human Cells Using the CRISPR-Cas9 System

(2013) Tim Wang et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Prediction of Deleterious Nonsynonymous Single-Nucleotide Polymorphism for Human Diseases

(2013) Jiaxin Wu et al.   TheScientificWorldJOURNAL

Double knockout pigs deficient in N-glycolylneuraminic acid and Galactose α-1,3-Galactose reduce the humoral barrier to xenotransplantation

(2013) Andrew J. Lutz et al.   XENOTRANSPLANTATION

Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus

(2013) Hirotaka Ebina et al.   Scientific Reports

Tunable and Multifunctional Eukaryotic Transcription Factors Based on CRISPR/Cas

(2013) Fahim Farzadfard et al.   ACS Synthetic Biology

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

Long-Term Rescue of Dystrophin Expression and Improvement in Muscle Pathology and Function in Dystrophic mdx Mice by Peptide-Conjugated Morpholino

(2012) Bo Wu et al.   AMERICAN JOURNAL OF PATHOLOGY

An Inhibitor of Nonhomologous End-Joining Abrogates Double-Strand Break Repair and Impedes Cancer Progression

(2012) Mrinal Srivastava et al.   CELL

Biological and Biomedical Applications of Engineered Nucleases

(2012) Yunzhi Pan et al.   MOLECULAR BIOTECHNOLOGY

RNA-guided genetic silencing systems in bacteria and archaea

(2012) Blake Wiedenheft et al.   NATURE

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas 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

Novel mechanisms of EBV-induced oncogenesis

(2012) Nancy Raab-Traub   Current Opinion in Virology

Double-Strand Break End Resection and Repair Pathway Choice

(2011) Lorraine S. Symington et al.   Annual Review of Genetics

Unification of Cas protein families and a simple scenario for the origin and evolution of CRISPR-Cas systems

(2011) Kira S Makarova et al.   Biology Direct

Generation of PPARγ mono-allelic knockout pigs via zinc-finger nucleases and nuclear transfer cloning

(2011) Dongshan Yang et al.   CELL RESEARCH

Challenges and Future Expectations of Reversed Gene Therapy

(2011) Nongyue He et al.   JOURNAL OF NANOSCIENCE AND NANOTECHNOLOGY

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

(2011) Jeffrey B Carroll et al.   MOLECULAR THERAPY

Evolution and classification of the CRISPR–Cas systems

(2011) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

Generation of Rat Pancreas in Mouse by Interspecific Blastocyst Injection of Pluripotent Stem Cells

(2010) Toshihiro Kobayashi et al.   CELL

Porcine reproductive and respiratory syndrome virus entry into the porcine macrophage

(2010) W. Van Breedam et al.   JOURNAL OF GENERAL VIROLOGY

Gene targeting with zinc finger nucleases to produce cloned eGFP knockout pigs

(2010) Jeffrey J. Whyte et al.   MOLECULAR REPRODUCTION AND DEVELOPMENT

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Genome editing with engineered zinc finger nucleases

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

Genomewide Association Studies and Assessment of the Risk of Disease

(2010) Teri A. Manolio   NEW ENGLAND JOURNAL OF MEDICINE

CRISPR/Cas, the Immune System of Bacteria and Archaea

(2010) P. Horvath et al.   SCIENCE

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo

(2009) Nicole K. Paulk et al.   HEPATOLOGY

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

(2009) J. Boch et al.   SCIENCE

Zinc-finger Nucleases: The Next Generation Emerges

(2008) Toni Cathomen et al.   MOLECULAR THERAPY