(Q45884500)

17 December 2017

title

In vivo gene therapy potentials of CRISPR-Cas9. (English)

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17 December 2017

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H-Y Xue

1

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17 December 2017

X Zhang

2

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17 December 2017

Y Wang

3

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17 December 2017

L Xiaojie

4

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17 December 2017

W-J Dai

5

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17 December 2017

Y Xu

6

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17 December 2017

publication date

31 March 2016

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17 December 2017

published in

Gene Therapy

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17 December 2017

volume

23

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17 December 2017

issue

7

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17 December 2017

page(s)

557-559

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17 December 2017

https://scigraph.springernature.com/pub.10.1038/gt.2016.25

exact match

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cites work

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RNA-guided human genome engineering via Cas9

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Multiplex genome engineering using CRISPR/Cas systems

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

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

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

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CRISPR-Cas9: a new and promising player in gene therapy

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

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

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

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

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Engineering adeno-associated viruses for clinical gene therapy

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

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

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CRISPR technology for gene therapy

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

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Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells

21 January 2018

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

21 January 2018

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

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

21 January 2018

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Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

21 January 2018

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Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9.

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

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21 January 2018

Identifiers

DOI

10.1038/GT.2016.25

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17 December 2017

Dimensions Publication ID

1047592936

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17 December 2017