ELSEVIER Critical Reviews in Oncology/Hematology 22 (1996) 61-78 Critical Reviews in ONCOLOGY/ HEMA TOLOG Y Stem cells from bone marrow, umbilical cord blood and peripheral blood for clinical application: current status and future application Li Lu*~*~, Rong-Nian Shenavcyd, Hal E. BroxmeyeraTbTd ‘Department of Medicine (Hematology/Oncology), Indiano University School of Medicine, Indianapolis, IN 46202-5321. USA bDepartment of Microbiology/Immunology, Indiano University School of Medicine, Indianapolis, IN 46202-5121, USA ‘Department of Radbtion Oncology, Indiano University School of Medicine, Indianapolis, IN 46202-5121, USA dThe Walther Oncology Center, Indiana University School of Medicine, Indianapolis, IN 46202-5121. USA Accepted 28 October 1994 Contents I. 2. 3. 4. 5. 6. 7. 8. Introduction .......................................................................... Hematopoietic stem cell purification and ex vivo expansion .................................. 2.1. Stem cell purification ............................................................. 2.2. Long-term culture and ex vivo expansion ........................................... Bone marrow stem cell transplantation .................................................... Umbilical cord blood stem cell transplantation ............................................. 4. I. Quantity and quality of immature hematopoietic stem cells from umbilical cord blood .... 4.2. Immune cells in cord blood ....................................................... 4.2.1. Number and phenotype of cord blood T-lymphocytes ......................... 4.2.2. Function of the T-lymphocytes in cord blood ................................ 4.2.3. Maternal cell contamination in cord blood ................................... Peripheral blood stem cell transplantation ................................................. 5.1. Blood stem cell mobilization and collection .......................................... 5.2. Kinetics of hemopoietic reconstitution after ABSCT .................................. 5.3. Contamination of harvested blood tumor cells ..................................... 5.4. Stability and sustainment of short- and long-term hemopoietic reconstitution after ABSC T Fetal liver stem cells (FLSC) transplantation ............................................ In utero hematopoietic stem cell transplants for inherited diseases .......................... Stem cell as target for gene therapy .................................................... 8.1. Increase transduction efficiency .................................................. 8.2. Long-term gene expression in hematopoietic cells in vivo ............................ 9. Conclusion ............................................................................ Acknowledgements .............................................................................. Reviewer ....................................................................................... References ..................................................................................... Biographies ..................................................................................... 62 62 62 63 64 65 65 65 66 66 66 66 67 68 68 68 69 69 69 70 70 71 71 71 71 78 Abbreviations: ABMT, autologous bone marrow transplantation; ABSCT, autologous blood stem cell transplantation; ADA, adenosine deaminase; ALL, acute. lymphocytic leukemia; allo, allogeneic; AML, acute myeloid leukemia; BM, bone marrow; BMT, bone marrow transplantation; CB, cord blood, CBSC, cord blood stem cells; CBSCT, cord blood stem cell transplantation; CML, chronic myeloid leukemia; CSF, colony stimulating factors; CT, cytotoxic therapy; FL, fetal liver; FLSC, fetal liver stem cells; GM-CSF, granulocyte macrophage-colony stimulating factor; GVHD, graft-versus-host disease; HGF, hematopoietic growth factors; HLA, human leukocyte antigens; HPP-CFC, high proliferative potential-colony for- ming cells; HSC, hematopoietic stem cells; IL, interleukin; LTC-IC, long-term culture-initiating cells; NK, natural killer; PB, peripheral blood; PBSC, peripheral blood stem cells; PCR, polymerase chain reaction; rhu, recombinant human; SCID, severe combined immunodeficiency disease; SLF, steel factor (also termed stem cell factor). *Corresponding author, Indiana University School of Medicine, 975 W. Walnut Street, Room 501,Indianapolis, IN 46202-5 121,USA. Tel.: (317) 274-7560; fax: (317) 274-7592. 1040-8428/96/$32.000 1996Elsevier Science Ireland Ltd. All rights reserved SSDI 1040-8428(95)0179-B