(Q39227008)

10 September 2017

Refining strategies to translate genome editing to the clinic (English)

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10 September 2017

3

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10 September 2017

1

Tatjana I Cornu

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10 September 2017

2

Claudio Mussolino

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PubMed ID

28388605

10 September 2017

language of work or name

English

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publication date

1 April 2017

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10 September 2017

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10 September 2017

23

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10 September 2017

4

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10 September 2017

415-423

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https://scigraph.springernature.com/pub.10.1038/nm.4313

10 September 2017

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cites work

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7 January 2021

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

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7 January 2021

Therapeutic genome editing: prospects and challenges

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7 January 2021

Genome Engineering With Zinc-Finger Nucleases

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7 January 2021

TALENs: a widely applicable technology for targeted genome editing

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7 January 2021

Gene therapy returns to centre stage

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7 January 2021

Altering the genome by homologous recombination

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7 January 2021

Expression of a site-specific endonuclease stimulates homologous recombination in mammalian cells

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7 January 2021

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

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7 January 2021

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

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7 January 2021

Targeted genome editing in human repopulating haematopoietic stem cells

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7 January 2021

Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease

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7 January 2021

Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

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7 January 2021

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.

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7 January 2021

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

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7 January 2021

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

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7 January 2021

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

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7 January 2021

Multiplex Genome-Edited T-cell Manufacturing Platform for "Off-the-Shelf" Adoptive T-cell Immunotherapies

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7 January 2021

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells.

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7 January 2021

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Small molecules enhance CRISPR genome editing in pluripotent stem cells.

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7 January 2021

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

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7 January 2021

Gene therapy on the move

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7 January 2021

In vivo genome editing restores haemostasis in a mouse model of haemophilia

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

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7 January 2021

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

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases

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7 January 2021

Treatment of ocular disorders by gene therapy.

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7 January 2021

Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy.

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Intracellular DNA recognition

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7 January 2021

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.

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7 January 2021

Adenoviral vector DNA for accurate genome editing with engineered nucleases.

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7 January 2021

mRNA transfection of a novel TAL effector nuclease (TALEN) facilitates efficient knockout of HIV co-receptor CCR5.

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7 January 2021

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

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7 January 2021

Chemical Biology Approaches to Genome Editing: Understanding, Controlling, and Delivering Programmable Nucleases

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7 January 2021

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

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Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles.

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7 January 2021

Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures

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Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion.

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7 January 2021

Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases

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7 January 2021

An improved zinc-finger nuclease architecture for highly specific genome editing

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7 January 2021

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

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7 January 2021

Genome editing with engineered zinc finger nucleases

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7 January 2021

Improved specificity of TALE-based genome editing using an expanded RVD repertoire

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7 January 2021

Optimized tuning of TALEN specificity using non-conventional RVDs.

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7 January 2021

Broad specificity profiling of TALENs results in engineered nucleases with improved DNA-cleavage specificity

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7 January 2021

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

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7 January 2021

Multiplex genome engineering using CRISPR/Cas systems

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7 January 2021

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

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7 January 2021

RNA-programmed genome editing in human cells

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7 January 2021

RNA-guided human genome engineering via Cas9

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7 January 2021

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

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7 January 2021

Cas9 as a versatile tool for engineering biology

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7 January 2021

Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases

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7 January 2021

Rationally engineered Cas9 nucleases with improved specificity

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7 January 2021

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

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7 January 2021

In vivo genome editing using Staphylococcus aureus Cas9

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7 January 2021

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

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7 January 2021

The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells

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7 January 2021

Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells.

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7 January 2021

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

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7 January 2021

Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects

1 reference

7 January 2021

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

1 reference

7 January 2021

Generation of TALE nickase-mediated gene-targeted cows expressing human serum albumin in mammary glands.

1 reference

7 January 2021

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

1 reference

7 January 2021

Genome engineering with targetable nucleases

1 reference

7 January 2021

CRISPR-Cas systems for editing, regulating and targeting genomes

1 reference

7 January 2021

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

1 reference

7 January 2021

Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing

1 reference

7 January 2021

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

1 reference

7 January 2021

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

1 reference

7 January 2021

Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome Editing

1 reference

7 January 2021

Chance or necessity? Insertional mutagenesis in gene therapy and its consequences

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7 January 2021

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity.

1 reference

7 January 2021

A TALE nuclease architecture for efficient genome editing

1 reference

7 January 2021

TALE nucleases: tailored genome engineering made easy

1 reference

7 January 2021

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

1 reference

7 January 2021

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

1 reference

7 January 2021

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

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7 January 2021

STAT1 and STAT3 in tumorigenesis: A matter of balance.

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7 January 2021

Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.

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7 January 2021

Zinc finger nucleases targeting the human papillomavirus E7 oncogene induce E7 disruption and a transformed phenotype in HPV16/18-positive cervical cancer cells

1 reference

7 January 2021

Inactivation of hepatitis B virus replication in cultured cells and in vivo with engineered transcription activator-like effector nucleases.

1 reference

7 January 2021

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

1 reference

7 January 2021

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

1 reference

7 January 2021

CRISPR-Cas9 mediated efficient PD-1 disruption on human primary T cells from cancer patients

1 reference

7 January 2021

A Multidrug-resistant Engineered CAR T Cell for Allogeneic Combination Immunotherapy

1 reference

7 January 2021

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

1 reference

7 January 2021

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

1 reference

7 January 2021

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

1 reference

7 January 2021

Identifiers

DOI

10.1038/NM.4313

1 reference

PubMed ID

28388605

Dimensions Publication ID

10 September 2017

0 references

1 reference

PubMed ID

28388605