(Q45873054)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29021165%20AND%20SRC:MED&resulttype=core&format=json

6 April 2020

title

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

1 reference

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6 April 2020

15

1 reference

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6 April 2020

Angelo Leone Lombardo

17

1 reference

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6 April 2020

Giulia Schiroli

1

1 reference

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6 April 2020

Samuele Ferrari

2

1 reference

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6 April 2020

Aurelien Jacob

4

1 reference

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6 April 2020

Valentina Capo

5

1 reference

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6 April 2020

Tiziana Plati

7

1 reference

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6 April 2020

Maria Carmina Castiello

8

1 reference

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6 April 2020

Rahul Palchaudhuri

12

1 reference

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6 April 2020

David T Scadden

13

1 reference

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6 April 2020

Giovanni Sitia

16

1 reference

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6 April 2020

Pietro Genovese

18

1 reference

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6 April 2020

Luigi Naldini

19

1 reference

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6 April 2020

author name string

Anthony Conway

3

1 reference

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6 April 2020

Luisa Albano

6

1 reference

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6 April 2020

Francesca Sanvito

9

1 reference

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6 April 2020

Andrew R Gennery

10

1 reference

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6 April 2020

Chiara Bovolenta

11

1 reference

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6 April 2020

Michael C Holmes

14

1 reference

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6 April 2020

language of work or name

English

0 references

publication date

1 October 2017

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29021165%20AND%20SRC:MED&resulttype=core&format=json

Science Translational Medicine

6 April 2020

published in

1 reference

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6 April 2020

volume

9

1 reference

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6 April 2020

issue

411

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29021165%20AND%20SRC:MED&resulttype=core&format=json

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

6 April 2020

cites work

1 reference

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

21 January 2018

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

21 January 2018

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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

21 January 2018

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

21 January 2018

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Targeted genome editing in human repopulating haematopoietic stem cells

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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

21 January 2018

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Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

21 January 2018

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Defective lymphoid development in mice lacking expression of the common cytokine receptor gamma chain.

21 January 2018

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Lymphoid development in mice with a targeted deletion of the interleukin 2 receptor gamma chain.

21 January 2018

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Lin-Sca1+kit- bone marrow cells contain early lymphoid-committed precursors that are distinct from common lymphoid progenitors

21 January 2018

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Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

21 January 2018

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A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

21 January 2018

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Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

21 January 2018

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Chemokine treatment rescues profound T-lineage progenitor homing defect after bone marrow transplant conditioning in mice

21 January 2018

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Cell competition is a tumour suppressor mechanism in the thymus.

21 January 2018

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Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin.

21 January 2018

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A differentiation checkpoint limits hematopoietic stem cell self-renewal in response to DNA damage

21 January 2018

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Stem Cell-Specific Mechanisms Ensure Genomic Fidelity within HSCs and upon Aging of HSCs

21 January 2018

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Hematopoietic stem cell quiescence promotes error-prone DNA repair and mutagenesis

21 January 2018

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

21 January 2018

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CRISPR-Cas9 knockin mice for genome editing and cancer modeling

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAN0820

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

21 January 2018

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Site-specific integration and tailoring of cassette design for sustainable gene transfer.

21 January 2018

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Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.

21 January 2018

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Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency

21 January 2018

1 reference

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

21 January 2018

1 reference

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A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

21 January 2018

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Gene therapy returns to centre stage

21 January 2018

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Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA.

21 January 2018

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DNA targeting specificity of RNA-guided Cas9 nucleases

21 January 2018

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Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAN0820

Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome

21 January 2018

1 reference

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Platelets prevent IFN-alpha/beta-induced lethal hemorrhage promoting CTL-dependent clearance of lymphocytic choriomeningitis virus.

21 January 2018

1 reference

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

21 January 2018

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Multi-reporter selection for the design of active and more specific zinc-finger nucleases for genome editing

21 January 2018

1 reference

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10.1126/SCITRANSLMED.AAN0820

21 January 2018

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29021165%20AND%20SRC:MED&resulttype=core&format=json

6 April 2020

1 reference