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

[1]  F. Alt,et al.  WASP deficiency leads to global defects of directed leukocyte migration in vitro and in vivo , 2005, Journal of leukocyte biology.

[2]  P. Opolon,et al.  A lentiviral vector encoding the human Wiskott–Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice , 2005, Gene Therapy.

[3]  C. Frecha,et al.  Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences , 2005, Gene Therapy.

[4]  W. Vainchenker,et al.  Mechanisms of WASp-mediated hematologic and immunologic disease. , 2004, Blood.

[5]  T. Laín de Lera,et al.  Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

[6]  P. Doherty,et al.  Defects in T-cell-mediated immunity to influenza virus in murine Wiskott-Aldrich syndrome are corrected by oncoretroviral vector-mediated gene transfer into repopulating hematopoietic cells. , 2003, Blood.

[7]  E. Haddad,et al.  A defect in hematopoietic stem cell migration explains the nonrandom X-chromosome inactivation in carriers of Wiskott-Aldrich syndrome. , 2003, Blood.

[8]  A. Nienhuis,et al.  Functional correction of T cells derived from patients with the Wiskott–Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein , 2003, Gene Therapy.

[9]  F. Alt,et al.  Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice. , 2003, Blood.

[10]  Christof von Kalle,et al.  Side effects of retroviral gene transfer into hematopoietic stem cells. , 2003, Blood.

[11]  Adrian J. Thrasher,et al.  Wasp in immune-system organization and function , 2002, Nature Reviews Immunology.

[12]  Yasuhiko Ito,et al.  Mixed chimera status of 12 patients with Wiskott-Aldrich syndrome (WAS) after hematopoietic stem cell transplantation: evaluation by flow cytometric analysis of intracellular WAS protein expression. , 2002, Blood.

[13]  G. Dunn,et al.  Restoration of podosomes and chemotaxis in Wiskott-Aldrich syndrome macrophages following induced expression of WASp. , 2002, The international journal of biochemistry & cell biology.

[14]  F. Candotti,et al.  Retrovirus-mediated WASP gene transfer corrects Wiskott-Aldrich syndrome T-cell dysfunction. , 2002, Human gene therapy.

[15]  Sheila M. Thomas,et al.  N-WASP deficiency reveals distinct pathways for cell surface projections and microbial actin-based motility , 2001, Nature Cell Biology.

[16]  G. E. Jones,et al.  Configuration of human dendritic cell cytoskeleton by Rho GTPases, the WAS protein, and differentiation. , 2001, Blood.

[17]  J. V. Stone,et al.  Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. , 2001, Blood.

[18]  C. Klein,et al.  Comparative Analysis of Genetically Modified Dendritic Cells and Tumor Cells as Therapeutic Cancer Vaccines , 2000, The Journal of experimental medicine.

[19]  C Kinnon,et al.  Wiskott-Aldrich syndrome protein is necessary for efficient IgG-mediated phagocytosis. , 2000, Blood.

[20]  S. Tsuboi,et al.  Expression of human Wiskott–Aldrich syndrome protein in patients’ cells leads to partial correction of a phenotypic abnormality of cell surface glycoproteins , 2000, Gene Therapy.

[21]  M. Aepfelbacher,et al.  Wiskott-Aldrich syndrome protein regulates podosomes in primary human macrophages. , 1999, Proceedings of the National Academy of Sciences of the United States of America.

[22]  F. Facchetti,et al.  Retrovirus-mediated WASP gene transfer corrects defective actin polymerization in B cell lines from Wiskott–Aldrich syndrome patients carrying ‘null’ mutations , 1999, Gene Therapy.

[23]  R. Mulligan,et al.  A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes. , 1996, Proceedings of the National Academy of Sciences of the United States of America.

[24]  A. Fischer,et al.  Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a single center. , 1996, The Journal of pediatrics.

[25]  J. James.  A MULTI-INSTITUTIONAL SURVEY OF THE WISKOTT-ALDRICH SYNDROME , 1996, Pediatrics.

[26]  U. Francke,et al.  Wiskott–Aldrich Syndrome Protein, a Novel Effector for the GTPase CDC42Hs, Is Implicated in Actin Polymerization , 1996, Cell.

[27]  F. Rosen,et al.  Nonrandom inactivation of the X chromosome in early lineage hematopoietic cells in carriers of Wiskott-Aldrich syndrome. , 1995, Blood.

[28]  K. Sullivan,et al.  A multiinstitutional survey of the Wiskott-Aldrich syndrome. , 1994, The Journal of pediatrics.

[29]  U. Francke,et al.  Isolation of a novel gene mutated in Wiskott-Aldrich syndrome , 1994, Cell.

[30]  J. Belli,et al.  Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone-marrow transplantation. , 1978, The New England journal of medicine.

[31]  L. Notarangelo,et al.  Primary immunodeficiency diseases , 2008 .

[32]  H. Ochs.  The Wiskott-Aldrich syndrome , 1998, Springer Seminars in Immunopathology.