(Q38757432)

6 September 2017

Therapeutic genome engineering via CRISPR-Cas systems (English)

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6 September 2017

0 references

1

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28198142%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

author name string

Prashant Mali

series ordinal

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28198142%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

publication date

15 February 2017

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Wiley interdisciplinary reviews. Systems biology and medicine

6 September 2017

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28198142%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

volume

9

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28198142%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

issue

4

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28198142%20AND%20SRC:MED&resulttype=core&format=json

Mechanism of homologous recombination: mediators and helicases take on regulatory functions

12 June 2020

cites work

1 reference

The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway

21 January 2018

1 reference

DNA recognition by Cys2His2 zinc finger proteins

21 January 2018

1 reference

Genome editing with engineered zinc finger nucleases

21 January 2018

1 reference

Genome Engineering With Zinc-Finger Nucleases

21 January 2018

1 reference

TALENs: a widely applicable technology for targeted genome editing

21 January 2018

1 reference

A large-scale in vivo analysis reveals that TALENs are significantly more mutagenic than ZFNs generated using context-dependent assembly

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

RNA-guided genetic silencing systems in bacteria and archaea

21 January 2018

1 reference

CRISPR/Cas, the immune system of bacteria and archaea

21 January 2018

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CRISPR Provides Acquired Resistance Against Viruses in Prokaryotes

21 January 2018

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

21 January 2018

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

21 January 2018

1 reference

Self versus non-self discrimination during CRISPR RNA-directed immunity.

21 January 2018

1 reference

RNA-programmed genome editing in human cells

21 January 2018

1 reference

RNA-guided human genome engineering via Cas9

21 January 2018

1 reference

Multiplex genome engineering using CRISPR/Cas systems

21 January 2018

1 reference

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

21 January 2018

1 reference

Effective gene targeting in rabbits using RNA-guided Cas9 nucleases.

21 January 2018

1 reference

Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing

21 January 2018

1 reference

CRISPR-Cas9 knockin mice for genome editing and cancer modeling

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

In vivo genome editing using Staphylococcus aureus Cas9

21 January 2018

1 reference

Inducible in vivo genome editing with CRISPR-Cas9

21 January 2018

1 reference

CRISPR/Cas9-induced disruption of gene expression in mouse embryonic brain and single neural stem cells in vivo.

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes

21 January 2018

1 reference

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

21 January 2018

1 reference

Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression

21 January 2018

1 reference

Highly efficient Cas9-mediated transcriptional programming

21 January 2018

1 reference

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

21 January 2018

1 reference

Repurposing the CRISPR-Cas9 system for targeted DNA methylation

21 January 2018

1 reference

A protein-tagging system for signal amplification in gene expression and fluorescence imaging

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

The CRISPR-associated DNA-cleaving enzyme Cpf1 also processes precursor CRISPR RNA.

21 January 2018

1 reference

Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells.

21 January 2018

1 reference

Generation of knockout mice by Cpf1-mediated gene targeting.

21 January 2018

1 reference

Progress and problems with the use of viral vectors for gene therapy

21 January 2018

1 reference

The use of retroviral vectors for gene therapy-what are the risks? A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery

21 January 2018

1 reference

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

21 January 2018

1 reference

Development and applications of CRISPR-Cas9 for genome engineering

21 January 2018

1 reference

Cas9 as a versatile tool for engineering biology

21 January 2018

1 reference

Phage response to CRISPR-encoded resistance in Streptococcus thermophilus

21 January 2018

1 reference

Diversity, activity, and evolution of CRISPR loci in Streptococcus thermophilus

21 January 2018

1 reference

Structure and Engineering of Francisella novicida Cas9.

21 January 2018

1 reference

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

21 January 2018

1 reference

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

21 January 2018

1 reference

Rational design of a split-Cas9 enzyme complex

21 January 2018

1 reference

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

21 January 2018

1 reference

Development of an intein-mediated split-Cas9 system for gene therapy

21 January 2018

1 reference

AAV-Mediated CRISPR/Cas Gene Editing of Retinal Cells In Vivo.

21 January 2018

1 reference

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

21 January 2018

1 reference

Genome-scale CRISPR-Cas9 knockout screening in human cells

21 January 2018

1 reference

Gene therapy returns to centre stage

21 January 2018

1 reference

Integration-deficient lentiviral vectors: a slow coming of age.

21 January 2018

1 reference

Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression in vitro and in vivo

21 January 2018

1 reference

Biosafety challenges for use of lentiviral vectors in gene therapy

21 January 2018

1 reference

Lentiviral vector gene therapy: effective and safe?

21 January 2018

1 reference

Non-viral vectors for gene-based therapy

21 January 2018

1 reference

Role of nanotechnology in targeted drug delivery and imaging: a concise review

21 January 2018

1 reference

In Vivo Delivery Systems for Therapeutic Genome Editing

21 January 2018

1 reference

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles.

21 January 2018

1 reference

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

21 January 2018

1 reference

Hydrodynamic gene delivery and its applications in pharmaceutical research

21 January 2018

1 reference

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

21 January 2018

1 reference

Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy

21 January 2018

1 reference

Virus binding to a plasma membrane receptor triggers interleukin-1 alpha-mediated proinflammatory macrophage response in vivo

21 January 2018

1 reference

Humoral and cellular immunity to an encoded protein induced by direct DNA injection

21 January 2018

1 reference

Liposome-mediated gene transfer into human vascular smooth muscle cells

21 January 2018

1 reference

A multifunctional AAV-CRISPR-Cas9 and its host response

21 January 2018

1 reference

DNA targeting specificity of RNA-guided Cas9 nucleases

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

21 January 2018

1 reference

sgRNAcas9: a software package for designing CRISPR sgRNA and evaluating potential off-target cleavage sites

21 January 2018

1 reference

Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

21 January 2018

1 reference

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

21 January 2018

1 reference

CRISPRscan: designing highly efficient sgRNAs for CRISPR-Cas9 targeting in vivo

21 January 2018

1 reference

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Rationally engineered Cas9 nucleases with improved specificity

21 January 2018

1 reference

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

21 January 2018

1 reference

Cas9-chromatin binding information enables more accurate CRISPR off-target prediction.

21 January 2018

1 reference

Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases.

21 January 2018

1 reference

Quantifying on- and off-target genome editing

21 January 2018

1 reference

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

21 January 2018

1 reference

A robust approach to identifying tissue-specific gene expression regulatory variants using personalized human induced pluripotent stem cells

21 January 2018

1 reference

Development of a mono-promoter-driven CRISPR/Cas9 system in mammalian cells.

21 January 2018

1 reference

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

21 January 2018

1 reference

High efficiency mutagenesis, repair, and engineering of chromosomal DNA using single-stranded oligonucleotides

21 January 2018

1 reference

A mechanism for the suppression of homologous recombination in G1 cells

21 January 2018

1 reference

Post-translational Regulation of Cas9 during G1 Enhances Homology-Directed Repair

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1002/WSBM.1380

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28198142%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

1 reference