(Q90788612)

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A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

scientific article published on 06 August 2018

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16 April 2020

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells (English)

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16 April 2020

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Christopher A Vakulskas

1

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16 April 2020

Garrett R Rettig

3

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16 April 2020

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16 April 2020

Ashley M Jacobi

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16 April 2020

Michael A Collingwood

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16 April 2020

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16 April 2020

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16 April 2020

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16 April 2020

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16 April 2020

Volker Wiebking

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16 April 2020

Natalia Gomez-Ospina

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16 April 2020

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16 April 2020

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16 April 2020

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Daniel P Dever

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16 April 2020

Matthew S McNeill

8

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16 April 2020

So Hyun Park

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16 April 2020

Mara Pavel-Dinu

16

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16 April 2020

Gang Bao

18

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16 April 2020

Matthew H Porteus

19

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16 April 2020

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6 August 2018

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16 April 2020

Nature Medicine

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16 April 2020

24

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16 April 2020

8

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16 April 2020

1216-1224

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16 April 2020

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

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

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7 January 2021

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Development and applications of CRISPR-Cas9 for genome engineering

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A conformational checkpoint between DNA binding and cleavage by CRISPR-Cas9

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Conformational control of DNA target cleavage by CRISPR-Cas9.

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Towards a new era in medicine: therapeutic genome editing

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The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation

1 reference

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7 January 2021

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Mapping the genomic landscape of CRISPR-Cas9 cleavage.

1 reference

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7 January 2021

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

1 reference

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7 January 2021

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Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9.

1 reference

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7 January 2021

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1 reference

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7 January 2021

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

1 reference

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7 January 2021

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

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7 January 2021

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A Cas9 Ribonucleoprotein Platform for Functional Genetic Studies of HIV-Host Interactions in Primary Human T Cells

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7 January 2021

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Generation of knock-in primary human T cells using Cas9 ribonucleoproteins.

1 reference

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7 January 2021

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

1 reference

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7 January 2021

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Immunological reconstitution of sex-linked lymphopenic immunological deficiency

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7 January 2021

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7 January 2021

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Gene therapy returns to centre stage

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7 January 2021

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CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease.

1 reference

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7 January 2021

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

1 reference

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7 January 2021

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Rationally engineered Cas9 nucleases with improved specificity

1 reference

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7 January 2021

based on heuristic

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

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

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Enhanced proofreading governs CRISPR-Cas9 targeting accuracy

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

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Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection.

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

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Engineered CRISPR-Cas9 nucleases with altered PAM specificities

1 reference

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7 January 2021

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A highly sensitive selection method for directed evolution of homing endonucleases

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7 January 2021

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RNase H-dependent PCR (rhPCR): improved specificity and single nucleotide polymorphism detection using blocked cleavable primers

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

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Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR.

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

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Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery.

1 reference

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7 January 2021

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

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7 January 2021

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

1 reference

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7 January 2021

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1 reference

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7 January 2021

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Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1.

1 reference

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7 January 2021

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A highly specific SpCas9 variant is identified by in vivo screening in yeast

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7 January 2021

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

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1 reference

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7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

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7 January 2021

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Pomalidomide reverses γ-globin silencing through the transcriptional reprogramming of adult hematopoietic progenitors

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7 January 2021

based on heuristic

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β-globin gene transfer to human bone marrow for sickle cell disease.

1 reference

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7 January 2021

based on heuristic

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1 reference

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7 January 2021

based on heuristic

inferred from DOI database lookup

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1 reference

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7 January 2021

based on heuristic

inferred from DOI database lookup

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA.

1 reference

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7 January 2021

based on heuristic

inferred from DOI database lookup

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing.

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Simplified CRISPR tools for efficient genome editing and streamlined protocols for their delivery into mammalian cells and mouse zygotes

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Use of inverse PCR to amplify and sequence breakpoints of HPRT deletion and translocation mutations.

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Non-nucleotide Modification of Anti-miRNA Oligonucleotides.

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Improvement of PCR reaction conditions for site-directed mutagenesis of big plasmids

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

crRNA and tracrRNA guide Cas9-mediated DNA interference in Streptococcus thermophilus

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

FLASH: fast length adjustment of short reads to improve genome assemblies

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

BEDTools: a flexible suite of utilities for comparing genomic features

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6.

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

AAV-mediated gene targeting methods for human cells

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Universal real-time PCR for the detection and quantification of adeno-associated virus serotype 2-derived inverted terminal repeat sequences

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

An efficient low cost method for gene transfer to T lymphocytes

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Easy quantitative assessment of genome editing by sequence trace decomposition

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

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7 January 2021

based on heuristic

inferred from DOI database lookup

A tale of three next generation sequencing platforms: comparison of Ion Torrent, Pacific Biosciences and Illumina MiSeq sequencers

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Genome editing assessment using CRISPR Genome Analyzer (CRISPR-GA).

1 reference

https://api.crossref.org/works/10.1038%2FS41591-018-0137-0

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1038/S41591-018-0137-0

1 reference

Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30082871%20AND%20SRC:MED&resulttype=core&format=json

16 April 2020

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30082871%20AND%20SRC:MED&resulttype=core&format=json

16 April 2020

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30082871%20AND%20SRC:MED&resulttype=core&format=json

16 April 2020

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