Developmental history and application of CRISPR in human disease (Q38669948)

5 September 2017

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title

Developmental history and application of CRISPR in human disease (English)

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5 September 2017

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4

Junjiu Huang

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author name string

Puping Liang

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1

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PubMed ID

28623876

5 September 2017

Xiya Zhang

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2

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PubMed ID

28623876

5 September 2017

Yuxi Chen

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3

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5 September 2017

17 June 2017

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5 September 2017

Journal of Gene Medicine

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5 September 2017

19

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5 September 2017

6-7

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Big Data: Astronomical or Genomical?

5 September 2017

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Gene targeting in mice: functional analysis of the mammalian genome for the twenty-first century

21 January 2018

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Establishment in culture of pluripotential cells from mouse embryos

21 January 2018

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Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells

21 January 2018

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Germline competent embryonic stem cells derived from rat blastocysts

21 January 2018

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Production of p53 gene knockout rats by homologous recombination in embryonic stem cells

21 January 2018

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Modeling disease in human ESCs using an efficient BAC-based homologous recombination system.

21 January 2018

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Sheep cloned by nuclear transfer from a cultured cell line

21 January 2018

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Viable offspring derived from fetal and adult mammalian cells

21 January 2018

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Full-term development of mice from enucleated oocytes injected with cumulus cell nuclei

21 January 2018

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Production of goats by somatic cell nuclear transfer

21 January 2018

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Cloned pigs produced by nuclear transfer from adult somatic cells

21 January 2018

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Cloned rabbits produced by nuclear transfer from adult somatic cells.

21 January 2018

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Generation of fertile cloned rats by regulating oocyte activation

21 January 2018

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Human factor IX transgenic sheep produced by transfer of nuclei from transfected fetal fibroblasts

21 January 2018

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Production of gene-targeted sheep by nuclear transfer from cultured somatic cells

21 January 2018

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Production of CFTR-null and CFTR-DeltaF508 heterozygous pigs by adeno-associated virus-mediated gene targeting and somatic cell nuclear transfer

21 January 2018

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Cloned transgenic calves produced from nonquiescent fetal fibroblasts

21 January 2018

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Production of alpha-1,3-galactosyltransferase knockout pigs by nuclear transfer cloning.

21 January 2018

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Targeted disruption of the alpha1,3-galactosyltransferase gene in cloned pigs

21 January 2018

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Functional disruption of the prion protein gene in cloned goats.

21 January 2018

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Production of cattle lacking prion protein.

21 January 2018

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Sequential targeting of the genes encoding immunoglobulin-mu and prion protein in cattle

21 January 2018

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Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease

21 January 2018

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Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae

21 January 2018

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

21 January 2018

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Stimulation of homologous recombination through targeted cleavage by chimeric nucleases

21 January 2018

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Genetic manipulation of genomes with rare-cutting endonucleases

21 January 2018

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The DNA Damage Response: Making It Safe to Play with Knives

21 January 2018

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

21 January 2018

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Gene targeting using zinc finger nucleases

21 January 2018

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Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains

21 January 2018

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Zinc finger-DNA recognition: crystal structure of a Zif268-DNA complex at 2.1 A

21 January 2018

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Selection of DNA binding sites for zinc fingers using rationally randomized DNA reveals coded interactions

21 January 2018

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DNA recognition of C2H2 zinc-finger proteins. Evidence for a zinc-finger-specific DNA recognition code

21 January 2018

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Rational design of artificial zinc-finger proteins using a nondegenerate recognition code table.

21 January 2018

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DNA recognition by Cys2His2 zinc finger proteins

21 January 2018

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Unexpected failure rates for modular assembly of engineered zinc fingers

21 January 2018

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A general strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites

21 January 2018

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A bacterial two-hybrid selection system for studying protein-DNA and protein-protein interactions

21 January 2018

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Targeted gene inactivation in zebrafish using engineered zinc-finger nucleases

21 January 2018

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Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification

21 January 2018

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Oligomerized pool engineering (OPEN): an 'open-source' protocol for making customized zinc-finger arrays

21 January 2018

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Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA)

21 January 2018

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An optimized two-finger archive for ZFN-mediated gene targeting

21 January 2018

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CRISPR-Cas systems for editing, regulating and targeting genomes

21 January 2018

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TALENs: a widely applicable technology for targeted genome editing

21 January 2018

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Xanthomonas AvrBs3 family-type III effectors: discovery and function

21 January 2018

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Breaking the code of DNA binding specificity of TAL-type III effectors

21 January 2018

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A simple cipher governs DNA recognition by TAL effectors

21 January 2018

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A guide to genome engineering with programmable nucleases

21 January 2018

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Targeting DNA double-strand breaks with TAL effector nucleases

21 January 2018

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An efficient strategy for TALEN-mediated genome engineering in Drosophila

21 January 2018

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Efficient targeted gene disruption in Xenopus embryos using engineered transcription activator-like effector nucleases (TALENs)

21 January 2018

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TALEN-mediated precise genome modification by homologous recombination in zebrafish.

21 January 2018

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A TALE nuclease architecture for efficient genome editing

21 January 2018

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High-efficiency TALEN-based gene editing produces disease-resistant rice.

21 January 2018

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Knockout rats generated by embryo microinjection of TALENs.

21 January 2018

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Efficient TALEN-mediated gene knockout in livestock

21 January 2018

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A library of TAL effector nucleases spanning the human genome

21 January 2018

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A TALEN genome-editing system for generating human stem cell-based disease models

21 January 2018

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RNA-guided human genome engineering via Cas9

21 January 2018

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RNA-programmed genome editing in human cells

21 January 2018

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Multiplex genome engineering using CRISPR/Cas systems

21 January 2018

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

21 January 2018

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Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system

21 January 2018

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Discovery and Functional Characterization of Diverse Class 2 CRISPR-Cas Systems

21 January 2018

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

21 January 2018

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Structures of Cas9 Endonucleases Reveal RNA-Mediated Conformational Activation

21 January 2018

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Crystal Structure of Staphylococcus aureus Cas9

21 January 2018

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Structure and Engineering of Francisella novicida Cas9.

21 January 2018

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Crystal Structure of Cpf1 in Complex with Guide RNA and Target DNA

21 January 2018

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The crystal structure of Cpf1 in complex with CRISPR RNA

21 January 2018

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Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product

21 January 2018

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Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements

21 January 2018

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Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin

21 January 2018

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CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies.

21 January 2018

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CRISPR Provides Acquired Resistance Against Viruses in Prokaryotes

21 January 2018

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21 January 2018

The Heroes of CRISPR.

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The discovery of CRISPR in archaea and bacteria

21 January 2018

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On the Origin of CRISPR-Cas Technology: From Prokaryotes to Mammals.

21 January 2018

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

21 January 2018

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A CRISPR approach to gene targeting

21 January 2018

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

21 January 2018

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

21 January 2018

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Multiplex and homologous recombination-mediated genome editing in Arabidopsis and Nicotiana benthamiana using guide RNA and Cas9.

21 January 2018

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

21 January 2018

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Precise and heritable genome editing in evolutionarily diverse nematodes using TALENs and CRISPR/Cas9 to engineer insertions and deletions

21 January 2018

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Genome editing with RNA-guided Cas9 nuclease in zebrafish embryos

21 January 2018

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Efficient genome editing in zebrafish using a CRISPR-Cas system

21 January 2018

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Efficient multiplex biallelic zebrafish genome editing using a CRISPR nuclease system

21 January 2018

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RNA-guided editing of bacterial genomes using CRISPR-Cas systems

21 January 2018

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Targeted genome modification of crop plants using a CRISPR-Cas system.

21 January 2018

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

21 January 2018

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One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering

21 January 2018

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Genome engineering in Saccharomyces cerevisiae using CRISPR-Cas systems

21 January 2018

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One-step generation of mice carrying reporter and conditional alleles by CRISPR/Cas-mediated genome engineering

21 January 2018

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Engineering the Caenorhabditis elegans genome using Cas9-triggered homologous recombination

21 January 2018

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Efficient nonmeiotic allele introgression in livestock using custom endonucleases

21 January 2018

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

21 January 2018

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DNA targeting specificity of RNA-guided Cas9 nucleases

21 January 2018

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High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

21 January 2018

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

21 January 2018

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Unexpected mutations after CRISPR–Cas9 editing in vivo

21 January 2018

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Mechanisms of oncogenic chromosomal translocations

21 January 2018

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Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity

21 January 2018

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Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects.

21 January 2018

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

21 January 2018

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

21 January 2018

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DNA-binding-domain fusions enhance the targeting range and precision of Cas9.

21 January 2018

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Rationally engineered Cas9 nucleases with improved specificity

21 January 2018

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

21 January 2018

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

21 January 2018

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Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

21 January 2018

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Targeted genome editing in human cells using CRISPR/Cas nucleases and truncated guide RNAs

21 January 2018

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

21 January 2018

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Optimizing sgRNA structure to improve CRISPR-Cas9 knockout efficiency.

21 January 2018

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An iCRISPR platform for rapid, multiplexable, and inducible genome editing in human pluripotent stem cells

21 January 2018

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

21 January 2018

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A split-Cas9 architecture for inducible genome editing and transcription modulation

21 January 2018

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Photoactivatable CRISPR-Cas9 for optogenetic genome editing

21 January 2018

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

21 January 2018

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Orthogonal Cas9 proteins for RNA-guided gene regulation and editing

21 January 2018

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In vivo genome editing using Staphylococcus aureus Cas9

21 January 2018

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Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications.

21 January 2018

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Efficient Production of Gene-Modified Mice using Staphylococcus aureus Cas9.

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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Nuclear domain 'knock-in' screen for the evaluation and identification of small molecule enhancers of CRISPR-based genome editing

21 January 2018

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

21 January 2018

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High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases

21 January 2018

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CRISPR/Cas9-based generation of knockdown mice by intronic insertion of artificial microRNA using longer single-stranded DNA

21 January 2018

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ssODN-mediated knock-in with CRISPR-Cas for large genomic regions in zygotes

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems.

21 January 2018

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Infection With Hepatitis C Virus Genotype 3 Is an Independent Risk Factor for End-Stage Liver Disease, Hepatocellular Carcinoma, and Liver-Related Death

21 January 2018

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Highly efficient RNA-guided base editing in mouse embryos.

21 January 2018

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Engineering and optimising deaminase fusions for genome editing

21 January 2018

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Generation of an ICF syndrome model by efficient genome editing of human induced pluripotent stem cells using the CRISPR system

21 January 2018

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

21 January 2018

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CRISPR-engineered mosaicism rapidly reveals that loss of Kcnj13 function in mice mimics human disease phenotypes

21 January 2018

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

21 January 2018

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Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer.

21 January 2018

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

21 January 2018

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CRISPR/Cas9-mediated Dax1 knockout in the monkey recapitulates human AHC-HH.

21 January 2018

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Cancer modeling in the modern era: progress and challenges

21 January 2018

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

21 January 2018

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Rapid modelling of cooperating genetic events in cancer through somatic genome editing

21 January 2018

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Simple and rapid in vivo generation of chromosomal rearrangements using CRISPR/Cas9 technology

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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Genome-scale CRISPR-Cas9 knockout screening in human cells

21 January 2018

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Genetic screens in human cells using the CRISPR-Cas9 system

21 January 2018

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

21 January 2018

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The promises and pitfalls of RNA-interference-based therapeutics

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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Fanconi anemia gene editing by the CRISPR/Cas9 system

21 January 2018

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CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells.

21 January 2018

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

21 January 2018

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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

21 January 2018

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Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

21 January 2018

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Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.

21 January 2018

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

21 January 2018

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Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients

21 January 2018

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Therapeutic genome editing: prospects and challenges

21 January 2018

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A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR.

21 January 2018

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Chinese scientists to pioneer first human CRISPR trial.

21 January 2018

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Biology of CCR5 and its role in HIV infection and treatment

21 January 2018

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

21 January 2018

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Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation

21 January 2018

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Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

21 January 2018

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Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection.

21 January 2018

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Zinc-finger nuclease editing of human cxcr4 promotes HIV-1 CD4(+) T cell resistance and enrichment

21 January 2018

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Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9

21 January 2018

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Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection.

21 January 2018

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CCR5 Disruption in Induced Pluripotent Stem Cells Using CRISPR/Cas9 Provides Selective Resistance of Immune Cells to CCR5-tropic HIV-1 Virus

21 January 2018

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

21 January 2018

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CCR5 gene disruption via lentiviral vectors expressing Cas9 and single guided RNA renders cells resistant to HIV-1 infection

21 January 2018

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CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity

21 January 2018

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

21 January 2018

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

21 January 2018

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Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing

21 January 2018

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Hepatitis B: the virus and disease

21 January 2018

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

21 January 2018

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Targeting Hepatitis B Virus With CRISPR/Cas9.

21 January 2018

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Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication.

21 January 2018

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Targeting hepatitis B virus cccDNA using CRISPR/Cas9.

21 January 2018

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Inhibition of hepatitis B virus by the CRISPR/Cas9 system via targeting the conserved regions of the viral genome

21 January 2018

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Harnessing the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated Cas9 system to disrupt the hepatitis B virus

21 January 2018

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

21 January 2018

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An Effective Molecular Target Site in Hepatitis B Virus S Gene for Cas9 Cleavage and Mutational Inactivation

21 January 2018

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Suppression of hepatitis B virus DNA accumulation in chronically infected cells using a bacterial CRISPR/Cas RNA-guided DNA endonuclease

21 January 2018

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CRISPR/Cas9-Derived Mutations Both Inhibit HIV-1 Replication and Accelerate Viral Escape.

21 January 2018

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CRISPR-Cas9 Can Inhibit HIV-1 Replication but NHEJ Repair Facilitates Virus Escape

21 January 2018

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Correction of a genetic disease in mouse via use of CRISPR-Cas9.

21 January 2018

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

21 January 2018

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Correction of the auditory phenotype in C57BL/6N mice via CRISPR/Cas9-mediated homology directed repair.

21 January 2018

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CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

21 January 2018

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Introducing precise genetic modifications into human 3PN embryos by CRISPR/Cas-mediated genome editing

21 January 2018

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

21 January 2018

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CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes

21 January 2018

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Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

21 January 2018

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Programmable RNA Tracking in Live Cells with CRISPR/Cas9.

21 January 2018

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Genome Editing: A New Approach to Human Therapeutics

21 January 2018

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Global summit reveals divergent views on human gene editing.

21 January 2018

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Biotechnology. A prudent path forward for genomic engineering and germline gene modification

21 January 2018

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Targetted correction of a mutant HPRT gene in mouse embryonic stem cells

21 January 2018

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21 January 2018

Identifiers

DOI

10.1002/JGM.2963

1 reference

5 September 2017

1 reference

PubMed ID

28623876