Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

R Zufferey, D Nagy, RJ Mandel, L Naldini… - Nature …, 1997 - nature.com
R Zufferey, D Nagy, RJ Mandel, L Naldini, D Trono
Nature biotechnology, 1997nature.com
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 …
Abstract
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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