CRISPR/Cas9‐mediated genome editing: From basic research to translational medicine

Genome-Scale CRISPRa Screen Identifies Novel Factors for Cellular Reprogramming

(2019) Jian Yang et al.   Stem Cell Reports

Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors

(2018) Silvana Konermann et al.   CELL

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos

(2018) Yanting Zeng et al.   MOLECULAR THERAPY

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

(2018) Johnny H. Hu et al.   NATURE

CRISPR/Cas9-mediated gene editing in human zygotes using Cas9 protein

(2017) Lichun Tang et al.   MOLECULAR GENETICS AND GENOMICS

RNA targeting with CRISPR–Cas13

(2017) Omar O. Abudayyeh et al.   NATURE

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Correction of a pathogenic gene mutation in human embryos

(2017) Hong Ma et al.   NATURE

Virus-Mediated Genome Editing via Homology-Directed Repair in Mitotic and Postmitotic Cells in Mammalian Brain

(2017) Jun Nishiyama et al.   NEURON

RNA editing with CRISPR-Cas13

(2017) David B. T. Cox et al.   SCIENCE

Nucleic acid detection with CRISPR-Cas13a/C2c2

(2017) Jonathan S. Gootenberg et al.   SCIENCE

Advancing chimeric antigen receptor T cell therapy with CRISPR/Cas9

(2017) Jiangtao Ren et al.   Protein & Cell

A decade of discovery: CRISPR functions and applications

(2017) Rodolphe Barrangou et al.   Nature Microbiology

Hypertrophic cardiomyopathy clinical phenotype is independent of gene mutation and mutation dosage

(2017) Shiv Kumar Viswanathan et al.   PLoS One

Biology and Applications of CRISPR Systems: Harnessing Nature’s Toolbox for Genome Engineering

(2016) Addison V. Wright et al.   CELL

Induced Pluripotent Stem Cells Meet Genome Editing

(2016) Dirk Hockemeyer et al.   Cell Stem Cell

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

(2016) Alexis C. Komor et al.   NATURE

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

(2016) Benjamin P. Kleinstiver et al.   NATURE

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA

(2016) Christopher D Richardson et al.   NATURE BIOTECHNOLOGY

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

(2016) Yang Yang 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

Comparison of Cas9 activators in multiple species

(2016) Alejandro Chavez et al.   NATURE METHODS

Cornerstones of CRISPR–Cas in drug discovery and therapy

(2016) Christof Fellmann et al.   NATURE REVIEWS DRUG DISCOVERY

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

(2016) Omar O. Abudayyeh et al.   SCIENCE

Advances in therapeutic CRISPR/Cas9 genome editing

(2016) Nataša Savić et al.   Translational Research

CRISPR-Cas9 for in vivo Gene Therapy: Promise and Hurdles

(2016) Wei-Jing Dai et al.   Molecular Therapy-Nucleic Acids

Phospholamban Ablation Using CRISPR/Cas9 System Improves Mortality in a Murine Heart Failure Model

(2016) Manami Kaneko et al.   PLoS One

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

(2015) M. D. Hoban et al.   BLOOD

High-Resolution CRISPR Screens Reveal Fitness Genes and Genotype-Specific Cancer Liabilities

(2015) Traver Hart et al.   CELL

Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9

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

Engineering Human Stem Cell Lines with Inducible Gene Knockout using CRISPR/Cas9

(2015) Yuejun Chen et al.   Cell Stem Cell

Recent developments and clinical studies utilizing engineered zinc finger nuclease technology

(2015) Young-Il Jo et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Adenovirus-Mediated Somatic Genome Editing ofPtenby CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

(2015) Dan Wang et al.   HUMAN GENE THERAPY

Dimeric CRISPR RNA-Guided FokI-dCas9 Nucleases Directed by Truncated gRNAs for Highly Specific Genome Editing

(2015) Nicolas Wyvekens et al.   HUMAN GENE THERAPY

Fanconi Anemia Gene Editing by the CRISPR/Cas9 System

(2015) Mark J. Osborn et al.   HUMAN GENE THERAPY

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

(2015) Yongchang Chen et al.   HUMAN MOLECULAR GENETICS

Inhibition of HIV-1 infection of primary CD4+ T-cells by gene editing of CCR5 using adenovirus-delivered CRISPR/Cas9

(2015) Chang Li et al.   JOURNAL OF GENERAL VIROLOGY

Expanding the Biologist’s Toolkit with CRISPR-Cas9

(2015) Samuel H. Sternberg et al.   MOLECULAR CELL

Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs

(2015) Cory Smith et al.   MOLECULAR THERAPY

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

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

Targeted disruption of DNMT1, DNMT3A and DNMT3B in human embryonic stem cells

(2015) Jing Liao et al.   NATURE GENETICS

Modeling colorectal cancer using CRISPR-Cas9–mediated engineering of human intestinal organoids

(2015) Mami Matano et al.   NATURE MEDICINE

Highly efficient Cas9-mediated transcriptional programming

(2015) Alejandro Chavez et al.   NATURE METHODS

Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements

(2015) Pratiksha I Thakore et al.   NATURE METHODS

High-throughput functional genomics using CRISPR–Cas9

(2015) Ophir Shalem et al.   NATURE REVIEWS GENETICS

An updated evolutionary classification of CRISPR–Cas systems

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

A genome-wide analysis of Cas9 binding specificity using ChIP-seq and targeted sequence capture

(2015) Henriette O'Geen et al.   NUCLEIC ACIDS RESEARCH

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins

(2015) Kathrin Schumann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker 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

Identification and characterization of essential genes in the human genome

(2015) T. Wang et al.   SCIENCE

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

(2015) David G. Ousterout et al.   Nature Communications

CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

(2015) Puping Liang et al.   Protein & Cell

Antibody-modified T cells: CARs take the front seat for hematologic malignancies

(2014) M. V. Maus et al.   BLOOD

MLL3 Is a Haploinsufficient 7q Tumor Suppressor in Acute Myeloid Leukemia

(2014) Chong Chen et al.   CANCER CELL

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

(2014) Hiroshi Nishimasu et al.   CELL

A Protein-Tagging System for Signal Amplification in Gene Expression and Fluorescence Imaging

(2014) Marvin E. Tanenbaum et al.   CELL

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9

(2014) Pankaj K. Mandal et al.   Cell Stem Cell

Low Incidence of Off-Target Mutations in Individual CRISPR-Cas9 and TALEN Targeted Human Stem Cell Clones Detected by Whole-Genome Sequencing

(2014) Adrian Veres et al.   Cell Stem Cell

Whole-Genome Sequencing Analysis Reveals High Specificity of CRISPR/Cas9 and TALEN-Based Genome Editing in Human iPSCs

(2014) Cory Smith et al.   Cell Stem Cell

An iCRISPR Platform for Rapid, Multiplexable, and Inducible Genome Editing in Human Pluripotent Stem Cells

(2014) Federico González et al.   Cell Stem Cell

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

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Efficient gene editing in adult mouse livers via adenoviral delivery of CRISPR/Cas9

(2014) Ranran Cheng et al.   FEBS LETTERS

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 andpiggyBac

(2014) Fei Xie et al.   GENOME RESEARCH

Chromosomal Translocations in Human Cells Are Generated by Canonical Nonhomologous End-Joining

(2014) Hind Ghezraoui et al.   MOLECULAR CELL

In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system

(2014) Danilo Maddalo et al.   NATURE

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9

(2014) Samuel H. Sternberg et al.   NATURE

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

Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease

(2014) Cem Kuscu et al.   NATURE BIOTECHNOLOGY

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

(2014) John P Guilinger 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

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Modeling the mitochondrial cardiomyopathy of Barth syndrome with induced pluripotent stem cell and heart-on-chip technologies

(2014) Gang Wang et al.   NATURE MEDICINE

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Evaluation of sgRNA Target Sites for CRISPR-Mediated Repression of TP53

(2014) Ingrid E. B. Lawhorn et al.   PLoS One

Protospacer Adjacent Motif (PAM)-Distal Sequences Engage CRISPR Cas9 DNA Target Cleavage

(2014) Regina Cencic 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

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Effective gene targeting in rabbits using RNA-guided Cas9 nucleases

(2014) Dongshan Yang et al.   Journal of Molecular Cell Biology

Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells

(2014) Luhan Yang et al.   Nature Communications

Engineering human tumour-associated chromosomal translocations with the RNA-guided CRISPR–Cas9 system

(2014) R. Torres et al.   Nature Communications

Targeted genomic rearrangements using CRISPR/Cas technology

(2014) Peter S. Choi et al.   Nature Communications

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

(2014) Steven Lin et al.   eLife

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

One-Step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-Mediated Genome Engineering

(2013) Hui Yang 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

Generation of gene-modified mice via Cas9/RNA-mediated gene targeting

(2013) Bin Shen et al.   CELL RESEARCH

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

(2013) Yuxuan Wu et al.   Cell Stem Cell

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

(2013) Qiurong Ding et al.   Cell Stem Cell

Cas9 effector-mediated regulation of transcription and differentiation in human pluripotent stem cells

(2013) N. A. Kearns et al.   DEVELOPMENT

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

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

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

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

(2013) Wei Li et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Heritable gene targeting in the mouse and rat using a CRISPR-Cas system

(2013) Dali Li et al.   NATURE BIOTECHNOLOGY

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

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

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

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Repair of Strand Breaks by Homologous Recombination

(2013) M. Jasin et al.   Cold Spring Harbor Perspectives in Biology

Playing the End Game: DNA Double-Strand Break Repair Pathway Choice

(2012) J. Ross Chapman et al.   MOLECULAR CELL

TALENs: a widely applicable technology for targeted genome editing

(2012) J. Keith Joung et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

(2011) Elitza Deltcheva et al.   NATURE

Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription

(2011) Feng Zhang et al.   NATURE BIOTECHNOLOGY

Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes

(2011) Ting Li et al.   NUCLEIC ACIDS RESEARCH

The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli

(2011) Rimantas Sapranauskas et al.   NUCLEIC ACIDS RESEARCH

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

(2010) Josiane E. Garneau et al.   NATURE

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

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

Mitotic homologous recombination maintains genomic stability and suppresses tumorigenesis

(2010) Mary Ellen Moynahan et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

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

(2009) J. Boch et al.   SCIENCE

A Simple Cipher Governs DNA Recognition by TAL Effectors

(2009) M. J. Moscou et al.   SCIENCE

Progress and prospects: Zinc-finger nucleases as gene therapy agents

(2008) D Carroll   GENE THERAPY

Small CRISPR RNAs Guide Antiviral Defense in Prokaryotes

(2008) S. J. J. Brouns et al.   SCIENCE

CRISPR Interference Limits Horizontal Gene Transfer in Staphylococci by Targeting DNA

(2008) Luciano A. Marraffini et al.   SCIENCE