Gene therapy: Targeting β-thalassaemia.

Nature ·Vol. 467 ·No. 7313 ·2010-09-16 ·Pages 277-8

Persons DA

Abstract

暂无摘要

MeSH Terms

Adolescent Blood Cells/cytology,metabolism Blood Transfusion Clone Cells/metabolism Genetic Therapy HMGA2 Protein/genetics,metabolism Humans Male Time Factors Transcriptional Activation Young Adult beta-Globins/genetics,metabolism beta-Thalassemia/genetics,metabolism,therapy

Chemicals

HMGA2 Protein beta-Globins

Authors & Affiliations

1 authors, click to expand affiliations / ORCID

Persons Derek A

References (10)

10 references, click to expand

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Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
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Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.
Science. 2002 Jun 28;296(5577):2410-3 PMID: 12089448
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.
Nature. 2010 Sep 16;467(7313):318-22 PMID: 20844535
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration.
Nat Biotechnol. 2006 Jun;24(6):687-96 PMID: 16732270
Roles of HMGA proteins in cancer.
Nat Rev Cancer. 2007 Dec;7(12):899-910 PMID: 18004397
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Science. 2003 Oct 17;302(5644):415-9 PMID: 14564000
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Blood. 2006 Dec 15;108(13):4232-6 PMID: 16940417
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.
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Article Info

Journal

Nature

Abbr.

Nature

ISSN

1476-4687

DOI

10.1038/467277a

Published

2010-09-16

Pages

277-8

Language

English

Region

England

NLM ID

0410462

Subset

Corrections

CommentOn

PMID: 20844535

External Links

PubMed source DOI: 10.1038/467277a View journal details

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Gene therapy: Targeting β-thalassaemia.

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