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Science 14 May 1993:
Vol. 260. no. 5110, pp. 926 - 932
DOI: 10.1126/science.8493530

Articles

Science, Vol 260, Issue 5110, 926-932
Copyright © 1993 by American Association for the Advancement of Science


articles

The basic science of gene therapy

RC Mulligan

Whitehead Institute for Biomedical Research, Massachusetts Institute of Technology, Cambridge 02142.

The development over the past decade of methods for delivering genes to mammalian cells has stimulated great interest in the possibility of treating human disease by gene-based therapies. However, despite substantial progress, a number of key technical issues need to be resolved before gene therapy can be safely and effectively applied in the clinic. Future technological developments, particularly in the areas of gene delivery and cell transplantation, will be critical for the successful practice of gene therapy.


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Gene transfer in the kidney.
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Development of a Self-Inactivating Lentivirus Vector.
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N. Uchida, R. E. Sutton, A. M. Friera, D. He, M. J. Reitsma, W. C. Chang, G. Veres, R. Scollay, and I. L. Weissman (1998)
PNAS 95, 11939-11944
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Hematopoietic Progenitor Cell Rolling in Bone Marrow Microvessels: Parallel Contributions by Endothelial Selectins and Vascular Cell Adhesion Molecule 1.
I. B. Mazo, J.-C. Gutierrez-Ramos, P. S. Frenette, R. O. Hynes, D. D. Wagner, and U. H. von Andrian (1998)
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Human Immunodeficiency Virus Type 1 Vectors Efficiently Transduce Human Hematopoietic Stem Cells.
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Multiple Sclerosis 4, 222-227
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Delayed Targeting of Cytokine-Nonresponsive Human Bone Marrow CD34+ Cells With Retrovirus-Mediated Gene Transfer Enhances Transduction Efficiency and Long-Term Expression of Transduced Genes.
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A combinatorial approach to the discovery of efficient cationic peptoid reagents for gene delivery.
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Transfection by Cationic Liposomes Using Simultaneous Single Cell Measurements of Plasmid Delivery and Transgene Expression.
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Liposome mediated gene transfer into the rat oesophagus.
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Vaccine Trials for the Clinician: Prospects for Viral and Non-Viral Vectors.
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Cell Cycle Analysis and Synchronization of Pluripotent Hematopoietic Progenitor Stem Cells.
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Optimal techniques for arterial gene transfer.
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Cardiovasc Res 35, 391-404
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Arterial gene transfer of acidic fibroblast growth factor for therapeutic angiogenesis in vivo: critical role of secretion signal in use of naked DNA.
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Cardiovasc Res 35, 470-479
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Immunosuppression prolongs adenoviral mediated transgene expression in cardiac allograft transplantation.
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Quantitative Analysis Reveals Expansion of Human Hematopoietic Repopulating Cells After Short-term Ex Vivo Culture.
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Major histocompatibility complex class II-transfected tumor cells present endogenous antigen and are potent inducers of tumor-specific immunity.
T. D. Armstrong, V. K. Clements, B. K. Martin, J. P.-Y. Ting, and S. Ostrand-Rosenberg (1997)
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Retroviral Gene Transduction of Adult Peripheral Blood or Marrow-Derived CD34+ Cells for Six Hours Without Growth Factors or on Autologous Stroma Does Not Improve Marking Efficiency Assessed In Vivo.
R.V.B. Emmons, S. Doren, J. Zujewski, M. Cottler-Fox, C.S. Carter, K. Hines, J.A. O'Shaughnessy, S.F. Leitman, J.J. Greenblatt, K. Cowan, et al. (1997)
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B. L. Maria, C. D. Medina, K. B.N. Hoang, and M. I. Phillips (1997)
J Child Neurol 12, 1-12
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Genetics: The Quiet Revolution in Science and Medicine: Implications for Research on Child Health Issues, Education of Health Professionals, and the New Preventive and Curative Medicine.
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