CRISPR-Cas9: a new and promising player in gene therapy (Q38364993)

2 September 2017

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title

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

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2 September 2017

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Lu Xiao-Jie

1

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2 September 2017

Xue Hui-Ying

2

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2 September 2017

Ke Zun-Ping

3

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2 September 2017

Chen Jin-Lian

4

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2 September 2017

Ji Li-Juan

5

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2 September 2017

language of work or name

English

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publication date

24 February 2015

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Journal of Medical Genetics

2 September 2017

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2 September 2017

52

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2 September 2017

5

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2 September 2017

289-296

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2 September 2017

Gene therapy on the move

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https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Gene therapy: Genie in a vector

21 January 2018

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https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Is cancer gene therapy an empty suit?

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Newer gene editing technologies toward HIV gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Pancreatic transduction by helper-dependent adenoviral vectors via intraductal delivery.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

The use of β-cell transcription factors in engineering artificial β cells from non-pancreatic tissue

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Gene-based therapies in Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

21 January 2018

History of gene therapy

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

A guide to genome engineering with programmable nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

A cut above the rest: targeted genome editing technologies in human pluripotent stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Multiplex genome engineering using CRISPR/Cas systems

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

RNA-guided human genome engineering via Cas9

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Genome editing. The new frontier of genome engineering with CRISPR-Cas9

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

A library of TAL effector nucleases spanning the human genome

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

TALEN or Cas9 - rapid, efficient and specific choices for genome modifications

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

CRISPR-Cas9 knockin mice for genome editing and cancer modeling

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Targeting Hepatitis B Virus With CRISPR/Cas9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Functional engraftment of colon epithelium expanded in vitro from a single adult Lgr5⁺ stem cell.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Low LDL cholesterol in individuals of African descent resulting from frequent nonsense mutations in PCSK9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

In vitro and in vivo growth suppression of human papillomavirus 16-positive cervical cancer cells by CRISPR/Cas9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

RNA-guided endonuclease provides a therapeutic strategy to cure latent herpesviridae infection

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Engineering complex synthetic transcriptional programs with CRISPR RNA scaffolds

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Synthesizing AND gate genetic circuits based on CRISPR-Cas9 for identification of bladder cancer cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Chromosomal translocations in human cells are generated by canonical nonhomologous end-joining.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Gene therapy and DNA delivery systems

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Directed evolution of novel adeno-associated viruses for therapeutic gene delivery.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Engineering adeno-associated viruses for clinical gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Orthogonal Cas9 proteins for RNA-guided gene regulation and editing

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Regulation of homologous recombination in eukaryotes

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Adenoviral vector DNA for accurate genome editing with engineered nucleases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

21 January 2018

Oncolytic virotherapy

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https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Clinical trials with oncolytic adenovirus in China

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Exploiting the curative potential of adoptive T-cell therapy for cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Dissecting mammalian immunity through mutation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Breakthrough of the year 2013. Cancer immunotherapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

Genome editing for human gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FJMEDGENET-2014-102968

10.1136/JMEDGENET-2014-102968

21 January 2018

Identifiers

DOI

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2 September 2017

1 reference