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Science 29 January 1999:
Vol. 283. no. 5402, pp. 682 - 686
DOI: 10.1126/science.283.5402.682
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Reports

Transduction of Human CD34+ Cells That Mediate Long-Term Engraftment of NOD/SCID Mice by HIV Vectors

Hiroyuki Miyoshi, * Kent A. Smith, * Donald E. Mosier, Inder M. Verma, dagger Bruce E. Torbett

Efficient gene transfer into human hematopoietic stem cells (HSCs) is an important goal in the study of the hematopoietic system as well as for gene therapy of hematopoietic disorders. A lentiviral vector based on the human immunodeficiency virus (HIV) was able to transduce human CD34+ cells capable of stable, long-term reconstitution of nonobese diabetic/severe combined immunodeficient (NOD/SCID) mice. High-efficiency transduction occurred in the absence of cytokine stimulation and resulted in transgene expression in multiple lineages of human hematopoietic cells for up to 22 weeks after transplantation.

H. Miyoshi and I. M. Verma, Laboratory of Genetics, Salk Institute for Biological Studies, La Jolla, CA 92037, USA. K. A. Smith, D. E. Mosier, B. E. Torbett, Department of Immunology, Scripps Research Institute, La Jolla, CA 92037, USA.
*   These authors contributed equally to this work.

dagger    To whom correspondence should be addressed. E-mail: verma{at}salk.edu

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Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein.
P. F. Kelly, J. Vandergriff, A. Nathwani, A. W. Nienhuis, and E. F. Vanin (2000)
Blood 96, 1206-1214
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Efficient transduction of human hematopoietic repopulating cells generating stable engraftment of transgene-expressing cells in NOD/SCID mice.
J. Barquinero, J. C. Segovia, M. Ramirez, A. Limon, G. Guenechea, T. Puig, J. Briones, J. Garcia, and J. A. Bueren (2000)
Blood 95, 3085-3093
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Maturation and Lineage-Specific Expression of the Coxsackie and Adenovirus Receptor in Hematopoietic Cells.
V. I. Rebel, S. Hartnett, J. Denham, M. Chan, R. Finberg, and C. A. Sieff (2000)
Stem Cells 18, 176-182
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A Murine Leukemia Virus (MuLV) Long Terminal Repeat Derived from Rhesus Macaques in the Context of a Lentivirus Vector and MuLV gag Sequence Results in High-Level Gene Expression in Human T Lymphocytes.
S. K. P. Kung, D. S. An, and I. S. Y. Chen (2000)
J. Virol. 74, 3668-3681
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Development of lentiviral vectors for gene therapy for human diseases.
G. L. Buchschacher Jr and F. Wong-Staal (2000)
Blood 95, 2499-2504
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Quantitative assessment of retroviral transfer of the human multidrug resistance 1 gene to human mobilized peripheral blood progenitor cells engrafted in nonobese diabetic/severe combined immunodeficient mice.
B. Schiedlmeier, K. Kuhlcke, H. G. Eckert, C. Baum, W. J. Zeller, and S. Fruehauf (2000)
Blood 95, 1237-1248
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Marking and Gene Expression by a Lentivirus Vector in Transplanted Human and Nonhuman Primate CD34+ Cells.
D. S. An, R. P. Wersto, B. A. Agricola, M. E. Metzger, S. Lu, R. G. Amado, I. S. Y. Chen, and R. E. Donahue (2000)
J. Virol. 74, 1286-1295
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Functional deletion of the CCR5 receptor by intracellular immunization produces cells that are refractory to CCR5-dependent HIV-1 infection and cell fusion.
P. Steinberger, J. Andris-Widhopf, B. Buhler, B. E. Torbett, and C. F. Barbas III (2000)
PNAS 97, 805-810
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Latest Developments in Gene Transfer Technology: Achievements, Perspectives, and Controversies over Therapeutic Applications.
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Stem Cells 18, 19-39
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Gene Therapy 2000.
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Hematology 2000, 376-393
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Primary Megakaryocytes Reveal a Role for Transcription Factor NF-E2 in Integrin {alpha}IIb{beta}3 Signaling.
M. Shiraga, A. Ritchie, S. Aidoudi, V. Baron, D. Wilcox, G. White, B. Ybarrondo, G. Murphy, A. Leavitt, and S. Shattil (1999)
J. Cell Biol. 147, 1419-1430
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Thymic Repopulation by CD34+ Human Cord Blood Cells After Expansion in Stroma-Free Culture.
B. Verhasselt, T. Kerre, E. Naessens, D. Vanhecke, M. De Smedt, B. Vandekerckhove, and J. Plum (1999)
Blood 94, 3644-3652
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A Lentivirus Packaging System Based on Alternative RNA Transport Mechanisms To Express Helper and Gene Transfer Vector RNAs and Its Use To Study the Requirement of Accessory Proteins for Particle Formation and Gene Delivery.
N. Srinivasakumar and F. G. Schuening (1999)
J. Virol. 73, 9589-9598
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Deficient Cellular Immunity--Finding and Fixing the Defects.
P. D. Greenberg and S. R. Riddell (1999)
Science 285, 546-551
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Clinical Trials Involving Multidrug Resistance Transcription Units in Retroviral Vectors.
A. B. Deisseroth (1999)
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Using HIV for Gene Therapy.
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Delivery of the Cre recombinase by a self-deleting lentiviral vector: Efficient gene targeting in vivo.
A. Pfeifer, E. P. Brandon, N. Kootstra, F. H. Gage, and I. M. Verma (2001)
PNAS 98, 11450-11455
   Abstract »    Full Text »    PDF »
Suppression of angiogenesis by lentiviral delivery of PEX, a noncatalytic fragment of matrix metalloproteinase 2.
A. Pfeifer, T. Kessler, S. Silletti, D. A. Cheresh, and I. M. Verma (2000)
PNAS 97, 12227-12232
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