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Journal of Virology, August 2001, p. 7030-7041, Vol. 75, No. 15
Department of Molecular Biology and
Genetics,1 Department of
Medicine,3 and Department of
Comparative Medicine and Pathology,4 The
Johns Hopkins University School of Medicine, Baltimore, Maryland
21205, and Heinrich-Pette-Institut für Experimentelle
Virologie und Immunologie an der Universität Hamburg, 20251 Hamburg, Germany2
Received 17 January 2001/Accepted 30 April 2001
Recently, remarkable progress has been made in developing effective
combination drug therapies that can control but not cure retroviral
replication. Even when effective, these drug regimens are toxic, they
require demanding administration schedules, and resistant viruses can
emerge. Thus the need for new gene-based therapies continues. In one
such approach, capsid-targeted viral inactivation (CTVI), nucleases
fused to viral coat proteins are expressed in infected cells and become
incorporated during virion assembly. CTVI can eliminate infectious
murine retrovirus titer in tissue culture. Here we describe transgenic
mice expressing fusions of the Moloney murine leukemia virus (Mo-MuLV)
Gag protein to staphylococcal nuclease. This work tests the protective
effect and demonstrates in vivo proof-of-principle of CTVI in
transgenic mice expressing endogenous proviral copies of Mo-MuLV. The
antiviral protein-expressing mice are phenotypically normal, attesting
to the lack of toxicity of the fusion protein. The Mo-MuLV infection was much less virulent in transgenic littermates than in nontransgenic littermates. Gag-nuclease expression reduced infectious titers in blood
up to 10-fold, decreased splenomegaly and leukemic infiltration, and
increased life spans up to 2.5-fold in transgenic relative to
nontransgenic infected animals. These results suggest that gene
therapies based on similar fusion proteins, designed to attack human
immunodeficiency virus or other retroviruses, could provide substantial
therapeutic benefits.
0022-538X/01/$04.00+0 DOI: 10.1128/JVI.75.15.7030-7041.2001
Copyright © 2001, American Society for Microbiology. All rights reserved.
Therapeutic Effect of a Gag-Nuclease Fusion
Protein against Retroviral Infection In Vivo

*
Corresponding author. Mailing address: Department of
Molecular Biology & Genetics, 725 North Wolfe St., 617 Hunterian Bldg., The Johns Hopkins University School of Medicine, Baltimore, MD 21205. Phone: (410) 955-0398. Fax: (410) 614-2987. E-mail:
jboeke{at}jhmi.edu.
Present address: Invitrogen Corp., Carlsbad, CA 92008.
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