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Research Article
Nature Biotechnology  15, 871 - 875 (1997)
doi:10.1038/nbt0997-871

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

Romain Zufferey1, 3, Dea Nagy2, Ron J. Mandel2, Luigi Naldini2 & Didier Trono1, 3, *

  1The Salk Institute, 10010 North Torrey Pines Road, La Jolla, CA 92037-1099.

  2Cell Genesys, Foster City, CA 94404.

  3Current address: Department of Genetics and Microbiology, CMU, 9 Avenue de Champel, CH-1211 Geneve 4, Switzerland.

  *e-mail: didier.trono@medecine.unige.ch.

Retroviral vectors derived from lentiviruses such as HIV-1 are promising tools for human gene therapy because they mediate the in vivo delivery and long-term expression of transgenes in nondividing tissues. We describe an HIV vector system in which the virulence genes env, vif, vpr, vpu, and nef have been deleted. This multiply attenuated vector conserved the ability to transduce growth-arrested cells and monocyte-derived macrophages in culture, and could efficiently deliver genes in vivo into adult neurons. These data demonstrate the potential of lentiviral vectors in human gene therapy.

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Nature Biotechnology
ISSN: 1087-0156
EISSN: 1546-1696
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