Development of lentiviral gene therapy for Wiskott Aldrich syndrome (Q56482184)

Expert Opinion on Biological Therapy

Europe PubMed Central

title

Development of lentiviral gene therapy for Wiskott Aldrich syndrome (English)

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Adrian J Thrasher

3

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author name string

Anne Galy

series ordinal

1

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Maria-Grazia Roncarolo

series ordinal

2

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language of work or name

English

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publication date

February 2008

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published in

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volume

8

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issue

2

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page(s)

181-90

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cites work

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Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.

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Interaction of the Wiskott-Aldrich syndrome protein with sorting nexin 9 is required for CD28 endocytosis and cosignaling in T cells

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A role for Wiskott-Aldrich syndrome protein in T-cell receptor-mediated transcriptional activation independent of actin polymerization

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WASP deficiency leads to global defects of directed leukocyte migration in vitro and in vivo

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Deficiency in the Wiskott-Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment

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Retroviral WASP gene transfer into human hematopoietic stem cells reconstitutes the actin cytoskeleton in myeloid progeny cells differentiated in vitro.

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Wiskott-Aldrich syndrome protein-deficient mice reveal a role for WASP in T but not B cell activation

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Antigen receptor-induced activation and cytoskeletal rearrangement are impaired in Wiskott-Aldrich syndrome protein-deficient lymphocytes

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Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells

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Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.

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Wiskott-Aldrich syndrome protein is involved in alphaIIb beta3-mediated cell adhesion

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A 5' regulatory sequence containing two Ets motifs controls the expression of the Wiskott-Aldrich syndrome protein (WASP) gene in human hematopoietic cells.

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