Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia (Q24618997)

Salima Hacein-Bey-Abina

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Laure Caccavelli

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Riccardo Sgarra

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Emmanuel Payen

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Philippe Leboulch

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Éliane Gluckman

Eliane Gluckman

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Olivier Negre

Olivier Negre

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Kathleen Hehir

Kathleen Hehir

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Floriane Fusil

Floriane Fusil

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Filippo Maria Denaro

Maria Denaro

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Troy Brady

Troy Brady

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Beatrix Gillet-Legrand

Beatrix Gillet-Legrand

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Stany Chretien

Stany Chrétien

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Patrick Aubourg

Patrick Aubourg

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Yves Beuzard

Yves Beuzard

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Nathalie Cartier

Nathalie Cartier

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Robert Girot

Robert Girot

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author name string

Gary Wang

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Julian Down

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Karen Westerman

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Resy Cavallesco

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Leila Maouche-Chrétien

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Françoise Bernaudin

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Ronald Dorazio

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Geert-Jan Mulder

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Axel Polack

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Arthur Bank

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Jean Soulier

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Jérôme Larghero

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Nabil Kabbara

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Bruno Dalle

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Bernard Gourmel

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Gérard Socie

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Kenneth Cornetta

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Frédéric Galacteros

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Frederick Bushman

The second AT-hook of the architectural transcription factor HMGA2 is determinant for nuclear localization and function

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language of work or name

English

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publication date

16 September 2010

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published in

Nature

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volume

467

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issue

7313

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page(s)

318-22

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cites work

1 reference

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MicroRNA maturation: stepwise processing and subcellular localization

7 April 2017

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

7 April 2017

1 reference

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Position-independent, high-level expression of the human beta-globin gene in transgenic mice

7 April 2017

1 reference

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Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

7 April 2017

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Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

27 September 2017

1 reference

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Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region

27 September 2017

1 reference

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Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

27 September 2017

1 reference

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Lin28 promotes transformation and is associated with advanced human malignancies

27 September 2017

1 reference

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Optimized lentiviral vector design improves titer and transgene expression of vectors containing the chicken beta-globin locus HS4 insulator element

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

27 September 2017

1 reference

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Studies in haemoglobin E beta-thalassaemia

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Long-term propagation of distinct hematopoietic differentiation programs in vivo

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

The HMGA proteins: a myriad of functions (Review).

27 September 2017

1 reference

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Lineage-specific transcription factors in multipotent hematopoietic progenitors: a little bit goes a long way.

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH).

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia

27 September 2017

1 reference

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Successful correction of the human Cooley's anemia beta-thalassemia major phenotype using a lentiviral vector flanked by the chicken hypersensitive site 4 chromatin insulator

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

The hematopoietic stem compartment consists of a limited number of discrete stem cell subsets

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Correction of sickle cell disease in transgenic mouse models by gene therapy

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Clinical and hematologic aspects of hemoglobin E beta-thalassemia

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

A 5' element of the chicken beta-globin domain serves as an insulator in human erythroid cells and protects against position effect in Drosophila

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Mapping of DNase I-hypersensitive sites in the upstream DNA of human embryonic epsilon-globin gene in K562 leukemia cells

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia

27 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3355472

Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells

30 May 2018

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FNATURE09328

Globin Lentiviral Vector Insertions Can Perturb the Expression of Endogenous Genes in β-thalassemic Hematopoietic Cells

21 January 2018

2 references

Crossref

https://api.crossref.org/works/10.1038%2FNATURE09328

21 January 2018

https://pubmed.ncbi.nlm.nih.gov/20844535

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Evidence that hematopoiesis may be a stochastic process in vivo

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FNATURE09328

21 January 2018

Identifiers

DOI

10.1038/NATURE09328

1 reference

Dimensions Publication ID

1049299780

0 references

1 reference

1 reference

PubMed publication ID

20844535

1 reference