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

[1]  Clelia Di Serio,et al.  Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration , 2006, Nature Biotechnology.

[2]  W. Vainchenker,et al.  A partial down-regulation of WASP is sufficient to inhibit podosome formation in dendritic cells. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

[3]  L. Naldini,et al.  Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation. , 2006, Human gene therapy.

[4]  R. Mulligan,et al.  Efficiency of transduction of highly purified murine hematopoietic stem cells by lentiviral and oncoretroviral vectors under conditions of minimal in vitro manipulation. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

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

[6]  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.

[7]  E. Medico,et al.  Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. , 2005, Blood.

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

[9]  H. Chou,et al.  Wiskott–Aldrich syndrome protein and the cytoskeletal dynamics of dendritic cells , 2004, The Journal of pathology.

[10]  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.

[11]  C. Heirman,et al.  Activation of monocytes via the CD14 receptor leads to the enhanced lentiviral transduction of immature dendritic cells. , 2004, Human gene therapy.

[12]  A. Thrasher,et al.  WASp deficiency in mice results in failure to form osteoclast sealing zones and defects in bone resorption. , 2004, Blood.

[13]  Wyeth W. Wasserman,et al.  JASPAR: an open-access database for eukaryotic transcription factor binding profiles , 2004, Nucleic Acids Res..

[14]  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.

[15]  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.

[16]  K. Siminovitch,et al.  The Wiskott–Aldrich syndrome protein: forging the link between actin and cell activation , 2003, Immunological reviews.

[17]  M. Aepfelbacher,et al.  Macrophages of patients with X‐linked thrombocytopenia display an attenuated Wiskott‐Aldrich syndrome phenotype , 2003, Immunology and cell biology.

[18]  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.

[19]  Michael J. Byrne,et al.  Mechanism of recruitment of WASP to the immunological synapse and of its activation following TCR ligation. , 2002, Molecular cell.

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

[21]  L. Dupré,et al.  Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation. , 2002, Immunity.

[22]  Wyeth W. Wasserman,et al.  TFBS: Computational framework for transcription factor binding site analysis , 2002, Bioinform..

[23]  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.

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

[25]  Kathryn L. Parsley,et al.  High-level transduction and gene expression in hematopoietic repopulating cells using a human imunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter , 2002 .

[26]  Kathryn L. Parsley,et al.  High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. , 2002, Human gene therapy.

[27]  D. Trono,et al.  High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. , 2000, Blood.

[28]  A. Thrasher,et al.  The Wiskott–Aldrich syndrome , 2000, Clinical and experimental immunology.

[29]  O. Danos,et al.  One step screening of retroviral producer clones by real time quantitative PCR , 1999, The journal of gene medicine.

[30]  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.

[31]  T. Hagemann,et al.  The identification and characterization of two promoters and the complete genomic sequence for the Wiskott-Aldrich syndrome gene. , 1999, Biochemical and biophysical research communications.

[32]  D. Trono,et al.  A Third-Generation Lentivirus Vector with a Conditional Packaging System , 1998, Journal of Virology.

[33]  I. Weissman,et al.  HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells. , 1998, Proceedings of the National Academy of Sciences of the United States of America.

[34]  A. Ballabio,et al.  A 5' regulatory sequence containing two Ets motifs controls the expression of the Wiskott-Aldrich syndrome protein (WASP) gene in human hematopoietic cells. , 1998, Blood.

[35]  J. Gutiérrez-Ramos,et al.  Identification of distinct elements of the stromal microenvironment that control human hematopoietic stem/progenitor cell growth and differentiation. , 1998, Experimental hematology.