(Q45865095)

17 December 2017

title

Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy (English)

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17 December 2017

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1

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17 December 2017

Sabata Martino

10

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17 December 2017

Alice Giustacchini

Alice Giustacchini

6

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Mario Amendola

Mario Amendola

8

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Angelo Quattrini

Angelo Quattrini

9

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John Dick

John E Dick

12

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Luigi Naldini

Luigi Naldini

14

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author name string

Ilaria Visigalli

2

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17 December 2017

Hidefumi Hiramatsu

3

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17 December 2017

Eric Lechman

4

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17 December 2017

Silvia Ungari

5

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17 December 2017

Giulia Schira

7

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17 December 2017

Aldo Orlacchio

11

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17 December 2017

Alessandra Biffi

13

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17 December 2017

language of work or name

English

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publication date

1 November 2010

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17 December 2017

Science Translational Medicine

published in

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17 December 2017

volume

2

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17 December 2017

issue

58

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17 December 2017

page(s)

58ra84

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17 December 2017

Hematopoietic stem-cell transplantation

cites work

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Gene therapy: therapeutic gene causing lymphoma

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Multilineage transcriptional priming and determination of alternate hematopoietic cell fates.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

MicroRNAs: target recognition and regulatory functions

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Small RNAs guide hematopoietic cell differentiation and function

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Molecular genetics of Krabbe disease (globoid cell leukodystrophy): Diagnostic and clinical implications

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

The endothelial-specific microRNA miR-126 governs vascular integrity and angiogenesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

miR-126 regulates angiogenic signaling and vascular integrity

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Stem cell concepts renew cancer research

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Identification of a hierarchy of multipotent hematopoietic progenitors in human cord blood

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

CD34+ hematopoietic stem-progenitor cell microRNA expression and function: a circuit diagram of differentiation control

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

A mammalian microRNA expression atlas based on small RNA library sequencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Comprehensive microRNA expression profiling of the hematopoietic hierarchy

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Regulation of progenitor cell proliferation and granulocyte function by microRNA-223

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

MicroRNA-126 regulates HOXA9 by binding to the homeobox

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Hox genes in hematopoiesis and leukemogenesis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

MicroRNAs in the Hox network: an apparent link to posterior prevalence

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

MicroRNA fingerprints during human megakaryocytopoiesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Regulation of angiogenesis through a microRNA (miR-130a) that down-regulates antiangiogenic homeobox genes GAX and HOXA5

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

Stable knockdown of microRNA in vivo by lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001522

10.1126/SCITRANSLMED.3001522

21 January 2018

Identifiers

DOI

1 reference

17 December 2017

1 reference

17 December 2017