Applications of CRISPR genome editing technology in drug target identification and validation

Non-kinase targets of protein kinase inhibitors

(2017) Lenka Munoz   NATURE REVIEWS DRUG DISCOVERY

Genomic targeting of epigenetic probes using a chemically tailored Cas9 system

(2017) Glen P. Liszczak et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

High content analysis platform for optimization of lipid mediated CRISPR-Cas9 delivery strategies in human cells

(2016) Benjamin Steyer et al.   Acta Biomaterialia

Improved delivery of Cas9 protein/gRNA complexes using lipofectamine CRISPRMAX

(2016) Xin Yu et al.   BIOTECHNOLOGY LETTERS

Identification of oncogenic driver mutations by genome-wide CRISPR-Cas9 dropout screening

(2016) Michael K. Kiessling et al.   BMC GENOMICS

Programmable RNA Tracking in Live Cells with CRISPR/Cas9

(2016) David A. Nelles et al.   CELL

CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs

(2016) Mohammad A. Mandegar et al.   Cell Stem Cell

A new class of temporarily phenotypic enhancers identified by CRISPR/Cas9-mediated genetic screening

(2016) Yarui Diao et al.   GENOME RESEARCH

Innovation in the pharmaceutical industry: New estimates of R&D costs

(2016) Joseph A. DiMasi et al.   JOURNAL OF HEALTH ECONOMICS

Genome editing in nonhuman primates: approach to generating human disease models

(2016) Y. Chen et al.   JOURNAL OF INTERNAL MEDICINE

Synthetic lethality in lung cancer and translation to clinical therapies

(2016) Ada W. Y. Leung et al.   Molecular Cancer

Methods for Optimizing CRISPR-Cas9 Genome Editing Specificity

(2016) Josh Tycko et al.   MOLECULAR CELL

Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome

(2016) Maximilian Müller et al.   MOLECULAR THERAPY

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

(2016) Dominik Paquet et al.   NATURE

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

(2016) Benjamin P. Kleinstiver et al.   NATURE

CRISPR knockout screening outperforms shRNA and CRISPRi in identifying essential genes

(2016) Bastiaan Evers et al.   NATURE BIOTECHNOLOGY

Systematic comparison of CRISPR/Cas9 and RNAi screens for essential genes

(2016) David W Morgens et al.   NATURE BIOTECHNOLOGY

Profiling of engineering hotspots identifies an allosteric CRISPR-Cas9 switch

(2016) Benjamin L Oakes et al.   NATURE BIOTECHNOLOGY

Multiplexed labeling of genomic loci with dCas9 and engineered sgRNAs using CRISPRainbow

(2016) Hanhui Ma et al.   NATURE BIOTECHNOLOGY

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

(2016) Christopher D Richardson et al.   NATURE BIOTECHNOLOGY

High-throughput mapping of regulatory DNA

(2016) Nisha Rajagopal et al.   NATURE BIOTECHNOLOGY

Functional genetic screens for enhancer elements in the human genome using CRISPR-Cas9

(2016) Gozde Korkmaz et al.   NATURE BIOTECHNOLOGY

Parallel shRNA and CRISPR-Cas9 screens enable antiviral drug target identification

(2016) Richard M Deans et al.   Nature Chemical Biology

Investigating essential gene function inMycobacterium tuberculosisusing an efficient CRISPR interference system

(2016) Atul K. Singh et al.   NUCLEIC ACIDS RESEARCH

Repurposing the CRISPR-Cas9 system for targeted DNA methylation

(2016) Aleksandar Vojta et al.   NUCLEIC ACIDS RESEARCH

Quantitative proteomics identify DAB2 as a cardiac developmental regulator that inhibits WNT/β-catenin signaling

(2016) Peter Hofsteen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Multiplexed barcoded CRISPR-Cas9 screening enabled by CombiGEM

(2016) Alan S. L. Wong 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

Disciplined approach to drug discovery and early development

(2016) Robert M. Plenge   Science Translational Medicine

Genomic Copy Number Dictates a Gene-Independent Cell Response to CRISPR/Cas9 Targeting

(2016) A. J. Aguirre et al.   Cancer Discovery

Systematic Cellular Disease Models Reveal Synergistic Interaction of Trisomy 21 and GATA1 Mutations in Hematopoietic Abnormalities

(2016) Kimihiko Banno et al.   Cell Reports

High-Content Analysis of CRISPR-Cas9 Gene-Edited Human Embryonic Stem Cells

(2016) Jared Carlson-Stevermer et al.   Stem Cell Reports

Epilepsy, Behavioral Abnormalities, and Physiological Comorbidities in Syntaxin-Binding Protein 1 (STXBP1) Mutant Zebrafish

(2016) Brian P. Grone et al.   PLoS One

When Quality Beats Quantity: Decision Theory, Drug Discovery, and the Reproducibility Crisis

(2016) Jack W. Scannell et al.   PLoS One

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

(2016) Yuichiro Miyaoka et al.   Scientific Reports

Engineering Complex Synthetic Transcriptional Programs with CRISPR RNA Scaffolds

(2015) Jesse G. Zalatan et al.   CELL

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

(2015) Sidi Chen et al.   CELL

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

(2015) Bernd Zetsche et al.   CELL

Small Molecules Enhance CRISPR Genome Editing in Pluripotent Stem Cells

(2015) Chen Yu et al.   Cell Stem Cell

Identifying Drug-Target Selectivity of Small-Molecule CRM1/XPO1 Inhibitors by CRISPR/Cas9 Genome Editing

(2015) Jasper E. Neggers et al.   CHEMISTRY & BIOLOGY

A CRISPR/Cas9 Vector System for Tissue-Specific Gene Disruption in Zebrafish

(2015) Julien Ablain et al.   DEVELOPMENTAL CELL

CRISPR/Cas9-mediatedDax1knockout in the monkey recapitulates human AHC-HH

(2015) Yu Kang et al.   HUMAN MOLECULAR GENETICS

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

(2015) Xiquan Liang et al.   JOURNAL OF BIOTECHNOLOGY

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

‘Organs-on-chips’ go mainstream

(2015) Sara Reardon   NATURE

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

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

(2015) Silvana Konermann et al.   NATURE

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

CRISPR-Cas9–based target validation for p53-reactivating model compounds

(2015) Michael Wanzel et al.   Nature Chemical Biology

The promise and peril of chemical probes

(2015) Cheryl H Arrowsmith et al.   Nature Chemical Biology

Large-scale identification of sequence variants influencing human transcription factor occupancy in vivo

(2015) Matthew T Maurano et al.   NATURE GENETICS

CAUSEL: an epigenome- and genome-editing pipeline for establishing function of noncoding GWAS variants

(2015) Sándor Spisák et al.   NATURE MEDICINE

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

(2015) Nicola A Kearns et al.   NATURE METHODS

Beyond editing: repurposing CRISPR–Cas9 for precision genome regulation and interrogation

(2015) Antonia A. Dominguez et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

FTO Obesity Variant Circuitry and Adipocyte Browning in Humans

(2015) Melina Claussnitzer et al.   NEW ENGLAND JOURNAL OF MEDICINE

Multicolor CRISPR labeling of chromosomal loci in human cells

(2015) Hanhui Ma et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Identification and characterization of essential genes in the human genome

(2015) T. Wang et al.   SCIENCE

Efficient inversions and duplications of mammalian regulatory DNA elements and gene clusters by CRISPR/Cas9

(2015) Jinhuan Li et al.   Journal of Molecular Cell Biology

Modelling kidney disease with CRISPR-mutant kidney organoids derived from human pluripotent epiblast spheroids

(2015) Benjamin S. Freedman et al.   Nature Communications

Synthesis of an arrayed sgRNA library targeting the human genome

(2015) Tobias Schmidt et al.   Scientific Reports

Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs

(2015) Amy L. Firth et al.   Cell Reports

Genome-wide CRISPR-Cas9 Screens Reveal Loss of Redundancy between PKMYT1 and WEE1 in Glioblastoma Stem-like Cells

(2015) Chad M. Toledo et al.   Cell Reports

Modeling of the Human Alveolar Rhabdomyosarcoma Pax3-Foxo1 Chromosome Translocation in Mouse Myoblasts Using CRISPR-Cas9 Nuclease

(2015) Irina V. Lagutina et al.   PLoS Genetics

A new age in functional genomics using CRISPR/Cas9 in arrayed library screening

(2015) Alexander Agrotis et al.   Frontiers in Genetics

Genome Editing and Its Applications in Model Organisms

(2015) Dongyuan Ma et al.   GENOMICS PROTEOMICS & BIOINFORMATICS

Morphological Profiles of RNAi-Induced Gene Knockdown Are Highly Reproducible but Dominated by Seed Effects

(2015) Shantanu Singh et al.   PLoS One

PCSK9: From discovery to therapeutic applications

(2014) Michel Farnier   Archives of Cardiovascular Diseases

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

(2014) Marvin E. Tanenbaum et al.   CELL

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

(2014) Luke A. Gilbert et al.   CELL

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu 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

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

(2014) Federico González et al.   Cell Stem Cell

OutKnocker: a web tool for rapid and simple genotyping of designer nuclease edited cell lines

(2014) Jonathan L. Schmid-Burgk et al.   GENOME RESEARCH

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

(2014) S. Kim et al.   GENOME RESEARCH

Saturation editing of genomic regions by multiplex homology-directed repair

(2014) Gregory M. Findlay et al.   NATURE

CRISPR-mediated direct mutation of cancer genes in the mouse liver

(2014) Wen Xue et al.   NATURE

Obesity-associated variants within FTO form long-range functional connections with IRX3

(2014) Scott Smemo et al.   NATURE

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

Microfluidic organs-on-chips

(2014) Sangeeta N Bhatia et al.   NATURE BIOTECHNOLOGY

Targeted sequencing by proximity ligation for comprehensive variant detection and local haplotyping

(2014) Paula J P de Vree et al.   NATURE BIOTECHNOLOGY

Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

(2014) Dirk Heckl 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

DNA sequencing and CRISPR-Cas9 gene editing for target validation in mammalian cells

(2014) Yegor Smurnyy et al.   Nature Chemical Biology

DrugTargetSeqR: a genomics- and CRISPR-Cas9–based method to analyze drug targets

(2014) Corynn Kasap et al.   Nature Chemical Biology

The discovery of first-in-class drugs: origins and evolution

(2014) Jörg Eder et al.   NATURE REVIEWS DRUG DISCOVERY

Investigating human disease using stem cell models

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

Targeting non-coding RNAs with the CRISPR/Cas9 system in human cell lines

(2014) Tsui-Ting Ho et al.   NUCLEIC ACIDS RESEARCH

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

Heritable Multiplex Genetic Engineering in Rats Using CRISPR/Cas9

(2014) Yuanwu Ma et al.   PLoS One

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

(2014) C. Long et al.   SCIENCE

Differential effects of P2Y1 deletion on glial activation and survival of photoreceptors and amacrine cells in the ischemic mouse retina

(2014) T Pannicke et al.   Cell Death & Disease

Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR–Cas platform

(2014) K. Yoshimi et al.   Nature Communications

Targeted genomic rearrangements using CRISPR/Cas technology

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

Multiplex genome engineering in human cells using all-in-one CRISPR/Cas9 vector system

(2014) Tetsushi Sakuma et al.   Scientific Reports

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

(2014) Katsuyuki Nakamura et al.   Scientific Reports

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

(2014) Steven Lin et al.   eLife

A CRISPR/Cas9-Based System for Reprogramming Cell Lineage Specification

(2014) Syandan Chakraborty et al.   Stem Cell Reports

Parallel genome-scale loss of function screens in 216 cancer cell lines for the identification of context-specific genetic dependencies

(2014) Glenn S Cowley et al.   Scientific Data

Beyond GWASs: Illuminating the Dark Road from Association to Function

(2013) Stacey L. Edwards et al.   AMERICAN JOURNAL OF HUMAN GENETICS

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

(2013) Haoyi Wang 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

Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system

(2013) Albert W Cheng et al.   CELL RESEARCH

Heritable gene-targeting with gRNA/Cas9 in rats

(2013) Xinli Hu et al.   CELL RESEARCH

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

(2013) Yuxuan Wu et al.   Cell Stem Cell

Modeling Human Disease with Pluripotent Stem Cells: from Genome Association to Function

(2013) Florian T. Merkle et al.   Cell Stem 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

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

(2013) Wei Li et al.   NATURE BIOTECHNOLOGY

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

(2013) Dali Li et al.   NATURE BIOTECHNOLOGY

Nucleotide-resolution DNA double-strand break mapping by next-generation sequencing

(2013) Nicola Crosetto et al.   NATURE METHODS

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

Heritable genome editing in C. elegans via a CRISPR-Cas9 system

(2013) Ari E Friedland et al.   NATURE METHODS

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

An integrated encyclopedia of DNA elements in the human genome

(2012)   NATURE

Diagnosing the decline in pharmaceutical R&D efficiency

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

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

(2012) M. Jinek et al.   SCIENCE

Systematic Localization of Common Disease-Associated Variation in Regulatory DNA

(2012) M. T. Maurano et al.   SCIENCE

Impact of high-throughput screening in biomedical research

(2011) Ricardo Macarron et al.   NATURE REVIEWS DRUG DISCOVERY

Toolkit for evaluating genes required for proliferation and survival using tetracycline-regulated RNAi

(2010) Johannes Zuber et al.   NATURE BIOTECHNOLOGY

Genetic Mapping in Human Disease

(2008) D. Altshuler et al.   SCIENCE