IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34+ cells while maintaining in vivo lymphoid potential.

[1]  M. Roncarolo,et al.  Mobilized blood CD34+ cells transduced and selected with a clinically applicable protocol reconstitute lymphopoiesis in SCID-Hu mice. , 2004, Human gene therapy.

[2]  Irving L Weissman,et al.  Biology of hematopoietic stem cells and progenitors: implications for clinical application. , 2003, Annual review of immunology.

[3]  C. Dunbar,et al.  Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. , 2003, Blood.

[4]  A. Fischer,et al.  Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99 , 2003, The Lancet.

[5]  A. Mortellaro,et al.  Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning , 2002, Science.

[6]  S. Karlsson,et al.  Efficient oncoretroviral transduction of extended long-term culture-initiating cells and NOD/SCID repopulating cells: enhanced reconstitution with gene-marked cells through an ex vivo expansion approach. , 2002, Human gene therapy.

[7]  L. Notarangelo,et al.  Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement , 2002, Nature Medicine.

[8]  A. Fischer,et al.  Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. , 2002, The New England journal of medicine.

[9]  F. Staal,et al.  Selective in vitro expansion and efficient retroviral transduction of human CD34+ CD38– haematopoietic stem cells , 2002, British journal of haematology.

[10]  M. Roncarolo,et al.  A novel human packaging cell line with hematopoietic supportive capacity increases gene transfer into early hematopoietic progenitors. , 2001, Human gene therapy.

[11]  T. Nakahata,et al.  Inhibitory effect of interleukin 3 on early development of human B‐lymphopoiesis , 2001, British journal of haematology.

[12]  D. Nelson,et al.  Somatic mosaicism in Wiskott–Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism , 2001, Proceedings of the National Academy of Sciences of the United States of America.

[13]  L. Barsky,et al.  Identification of a novel, human multilymphoid progenitor in cord blood. , 2001, Blood.

[14]  K. Yamaguchi,et al.  T-cell lines from 2 patients with adenosine deaminase (ADA) deficiency showed the restoration of ADA activity resulted from the reversion of an inherited mutation. , 2001, Blood.

[15]  Y. Hamel,et al.  Optimization of retroviral gene transfer protocol to maintain the lymphoid potential of progenitor cells. , 2001, Human gene therapy.

[16]  M. Roncarolo,et al.  Construction of Human‐SCID Chimeric Mice , 1998, Current protocols in immunology.

[17]  W. Piacibello,et al.  Negative influence of IL3 on the expansion of human cord blood in vivo long-term repopulating stem cells. , 2000, Journal of hematotherapy & stem cell research.

[18]  K Khare,et al.  Metabolites from apoptotic thymocytes inhibit thymopoiesis in adenosine deaminase-deficient fetal thymic organ cultures. , 2000, The Journal of clinical investigation.

[19]  L. Barsky,et al.  IL-3 Increases Production of B Lymphoid Progenitors from Human CD34+CD38− Cells1 , 2000, The Journal of Immunology.

[20]  J. Briones,et al.  Efficient transduction of human hematopoietic repopulating cells generating stable engraftment of transgene-expressing cells in NOD/SCID mice. , 2000, Blood.

[21]  F. Deist,et al.  Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. , 2000, Science.

[22]  Hiroshi Nakajima,et al.  Janus kinase 3 (Jak3) is essential for common cytokine receptor γ chain (γc)-dependent signaling: comparative analysis of γc, Jak3, and γc and Jak3 double-deficient mice , 2000 .

[23]  W. Leonard,et al.  Janus kinase 3 (Jak3) is essential for common cytokine receptor gamma chain (gamma(c))-dependent signaling: comparative analysis of gamma(c), Jak3, and gamma(c) and Jak3 double-deficient mice. , 2000, International immunology.

[24]  R. Hoffman,et al.  An in vivo competitive repopulation assay for various sources of human hematopoietic stem cells. , 2000, Blood.

[25]  J. Nolta,et al.  The number and generative capacity of human B lymphocyte progenitors, measured in vitro and in vivo, is higher in umbilical cord blood than in adult or pediatric bone marrow , 1999, Bone Marrow Transplantation.

[26]  Michael P. Brown,et al.  Reconstitution of early lymphoid proliferation and immune function in Jak3-deficient mice by interleukin-3. , 1999, Blood.

[27]  S. Forestell,et al.  Optimization of retroviral gene transduction of mobilized primitive hematopoietic progenitors by using thrombopoietin, Flt3, and Kit ligands and RetroNectin culture. , 1999, Human gene therapy.

[28]  W. Vainchenker,et al.  Identification of Lymphomyeloid Primitive Progenitor Cells in Fresh Human Cord Blood and in the Marrow of Nonobese Diabetic–Severe Combined Immunodeficient (NOD-SCID) Mice Transplanted with Human CD34+ Cord Blood Cells , 1999, The Journal of experimental medicine.

[29]  K. Moore,et al.  Single adult human CD34(+)/Lin-/CD38(-) progenitors give rise to natural killer cells, B-lineage cells, dendritic cells, and myeloid cells. , 1999, Blood.

[30]  C. Shih,et al.  Long-term ex vivo maintenance and expansion of transplantable human hematopoietic stem cells. , 1999, Blood.

[31]  Steven F. Ziegler,et al.  Defective IL7R expression in T-B+NK + severe combined immunodeficiency , 1998, Nature Genetics.

[32]  R. Schiffmann,et al.  Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation. , 1998, Human gene therapy.

[33]  F. Hirayama,et al.  Negative regulation by interleukin-3 (IL-3) of mouse early B-cell progenitors and stem cells in culture: transduction of the negative signals by betac and betaIL-3 proteins of IL-3 receptor and absence of negative regulation by granulocyte-macrophage colony-stimulating factor. , 1998, Blood.

[34]  Lesley J. Murray,et al.  Thrombopoietin, kit ligand, and flk2/flt3 ligand together induce increased numbers of primitive hematopoietic progenitors from human CD34+Thy-1+Lin- cells with preserved ability to engraft SCID-hu bone. , 1998, Blood.

[35]  I. Weissman,et al.  Identification of Clonogenic Common Lymphoid Progenitors in Mouse Bone Marrow , 1997, Cell.

[36]  M. Ogawa,et al.  Development of natural killer cells, B lymphocytes, macrophages, and mast cells from single hematopoietic progenitors in culture of murine fetal liver cells. , 1997, Blood.

[37]  I. Weissman,et al.  Bcl-2 Rescues T Lymphopoiesis in Interleukin-7 Receptor–Deficient Mice , 1997, Cell.

[38]  M. Roncarolo,et al.  Phenotypic and functional evidence for the expression of CD4 by hematopoietic stem cells isolated from human fetal liver. , 1997, Blood.

[39]  J. Liesveld,et al.  Expression of interleukin-7 receptor by lineage-negative human bone marrow progenitors with enhanced lymphoid proliferative potential and B-lineage differentiation capacity. , 1997, Blood.

[40]  F. Rieux-Laucat,et al.  Naturally occurring primary deficiencies of the immune system. , 1997, Annual review of immunology.

[41]  T. Lebien,et al.  Interleukin 7 independent development of human B cells. , 1996, Proceedings of the National Academy of Sciences of the United States of America.

[42]  R. Hoffman,et al.  High-level expression of a novel epitope of CD59 identifies a subset of CD34+ bone marrow cells highly enriched for pluripotent stem cells. , 1996, Experimental hematology.

[43]  J. Puck,et al.  Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency , 1996, Nature Genetics.

[44]  A. Fischer,et al.  Bone marrow gene transfer in three patients with adenosine deaminase deficiency. , 1996, Gene therapy.

[45]  F. Hirayama,et al.  Negative regulation of early T lymphopoiesis by interleukin-3 and interleukin-1 alpha. , 1995, Blood.

[46]  S. Rosenberg,et al.  T Lymphocyte-Directed Gene Therapy for ADA− SCID: Initial Trial Results After 4 Years , 1995, Science.

[47]  Evelina Mazzolari,et al.  Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA− Immunodeficient Patients , 1995, Science.

[48]  A. Galy,et al.  Human T, B, natural killer, and dendritic cells arise from a common bone marrow progenitor cell subset. , 1995, Immunity.

[49]  K. Weinberg,et al.  Engraftment of gene–modified umbilical cord blood cells in neonates with adenosine deaminase deficiency , 1995, Nature Medicine.

[50]  A. Nienhuis,et al.  Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. , 1995, Blood.

[51]  A. Galy,et al.  Engraftment of human hematopoietic precursor cells with secondary transfer potential in SCID-hu mice. , 1994, Blood.

[52]  R. de Waal Malefyt,et al.  High levels of interleukin 10 production in vivo are associated with tolerance in SCID patients transplanted with HLA mismatched hematopoietic stem cells , 1994, The Journal of experimental medicine.

[53]  S. Nishikawa,et al.  Expression and function of the interleukin 7 receptor in murine lymphocytes. , 1993, Proceedings of the National Academy of Sciences of the United States of America.

[54]  F. Finkelman,et al.  Inhibition of murine B and T lymphopoiesis in vivo by an anti- interleukin 7 monoclonal antibody , 1993, The Journal of experimental medicine.

[55]  A. Zlotnik,et al.  IL-7 maintains the T cell precursor potential of CD3-CD4-CD8- thymocytes. , 1991, Journal of immunology.