Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors

Nature Biotechnology

Volumn 23, Issue 6, 2005, Pages 709-717

  Song, Erwei ^a   Zhu, Pengcheng ^a   Lee, Sang Kyung ^a   Chowdhury, Dipanjan ^a   Kussman, Steven ^a   Dykxhoorn, Derek M ^a   Feng, Yi ^a   Palliser, Deborah ^a   Weiner, David B ^b   Shankar, Premlata ^a   Marasco, Wayne A ^a   Lieberman, Judy ^a  

^a HARVARD MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMINES; CELLS; RNA; TISSUE; VIRUSES;

FUSION PROTEIN; PROTAMINE CODING SEQUENCE;

ANTIBODIES;

ANTIBODY; CELL SURFACE RECEPTOR; GAG PROTEIN; HYBRID PROTEIN; MYC PROTEIN; PROTAMINE; PROTEIN MDM2; SMALL INTERFERING RNA; VASCULOTROPIN; INTERFERON; VIRUS RECEPTOR;

ANIMAL EXPERIMENT; ARTICLE; CARBOXY TERMINAL SEQUENCE; CELL PROLIFERATION; GENE EXPRESSION; GENE SILENCING; GENE TARGETING; HEAVY CHAIN; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IN VIVO STUDY; MELANOMA CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; RNA INTERFERENCE; VIRUS ENVELOPE; VIRUS REPLICATION; ANIMAL; CELL STRAIN COS1; CHEMISTRY; DRUG DELIVERY SYSTEM; EXPERIMENTAL MELANOMA; HUMAN; METABOLISM; PHYSIOLOGY;

ANIMALIA; HUMAN IMMUNODEFICIENCY VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1; MIRIDAE;

ANIMALS; ANTIBODIES; COS CELLS; DRUG DELIVERY SYSTEMS; HIV-1; HUMANS; INTERFERONS; MELANOMA, EXPERIMENTAL; MICE; RECEPTORS, CELL SURFACE; RECEPTORS, HIV; RECOMBINANT FUSION PROTEINS; RNA, SMALL INTERFERING;

EID: 23844474160     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt1101     Document Type: Article

References (31)

1. Dykxhoorn, D.M. & Lieberman, J. The Silent Revolution: RNA interference as basic biology, research tool and therapeutic. Annu. Rev. Med. 56, 401-423 (2005).
2. Sledz, C.A., Holko, M., de Veer, M.J., Silverman, R.H. & Williams, B.R. Activation of the interferon system by short-interfering RNAs. Nat. Cell Biol. 5, 834-839 (2003).
3. Jackson, A.L. et al. Expression profiling reveals off-target gene regulation by RNAi. Nat. Biotechnol. 21, 635-637 (2003).
4. Kariko, K., Bhuyan, P., Capodici, J. & Weissman, D. Small interfering RNAs mediate sequence-independent gene suppression and induce immune activation by signaling through toll-like receptor 3. J. Immunol. 172, 6545-6549 (2004).
5. Bitko, V., Musiyenko, A., Shulyayeva, O. & Barik, S. Inhibition of respiratory viruses by nasally administered siRNA. Nat. Med. 11, 50-55 (2005).
6. Song, E. et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat. Med. 9, 347-351 (2003).
7. Chen, S.Y., Khouri, Y., Bagley, J. & Marasco, W.A. Combined intra- and extracellular immunization against human immunodeficiency virus type 1 infection with a human anti-gp120 antibody. Proc. Natl. Acad. Sci. USA 91, 5932-5936 (1994).
8. Chen, S.Y., Zani, C., Khouri, Y. & Marasco, W.A. Design of a genetic immunotoxin to eliminate toxin immunogenicity. Gene Ther. 2, 116-123 (1995).
9. Chen, T.H. et al. Structure of pp32, an acidic nuclear protein which inhibits oncogene-induced formation of transformed foci. Mol. Biol. Cell 7, 2045-2056 (1996).
10. Li, X., Stuckert, P., Bosch, I., Marks, J.D. & Marasco, W.A. Single-chain antibody-mediated gene delivery into ErbB2-positive human breast cancer cells. Cancer Gene Ther. 8, 555-565 (2001).
11. Layzer, J.M. et al. In vivo activity of nuclease-resistant siRNAs. RNA 10, 766-771 (2004).
12. Soutschek, J. et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432, 173-178 (2004).
13. Song, E. et al. Sustained Small Interfering RNA-Mediated Human Immunodeficiency Virus Type 1 Inhibition in Primary Macrophages. J. Virol. 77, 7174-7181 (2003).
14. Slamon, D.J. et al. Human breast cancer: correlation of relapse and survival with amplification of the HER-2/neu oncogene. Science 235, 177-182 (1987).
15. Sorensen, D.R., Leirdal, M. & Sioud, M. Gene silencing by systemic delivery of synthetic siRNAs in adult mice. J. Mol. Biol. 327, 761-766 (2003).
16. Zhang, X. et al. Small interfering RNA targeting heme oxygenase-1 enhances ischemia-reperfusion-induced lung apoptosis. J. Biol. Chem. 279, 10677-10684 (2004).
17. Massaro, D., Massaro, G.D. & Clerch, L.B. Noninvasive delivery of small inhibitory RNA and other reagents to pulmonary alveoli in mice. Am. J. Physiol. Lung Cell. Mol. Physiol. 287, L1066-L1070 (2004).
18. McCaffrey, A.P. et al. Inhibition of hepatitis B virus in mice by RNA interference. Nat. Biotechnol. 21, 639-644 (2003).
19. McCaffrey, A.P. et al. RNA interference in adult mice. Nature 418, 38-39 (2002).
20. Hamar, P. et al. Small interfering RNA targeting Fas protects mice against renal ischemia-reperfusion injury. Proc. Natl. Acad. Sci. USA 101, 14883-14888 (2004).
21. Contreras, J.L. et al. Caspase-8 and caspase-3 small interfering RNA decreases ischemia/reperfusion injury to the liver in mice. Surgery 136, 390-400 (2004).
22. Minakuchi, Y. et al. Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo. Nucleic Acids Res. 32, e109 (2004).
23. Schiffelers, R.M. et al. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic Acids Res. 32, e149 (2004).
24. Hassani, Z. et al. Lipid-mediated siRNA delivery down-regulates exogenous gene expression in the mouse brain at picomolar levels. J. Gene Med. (2004).
25. Ge, Q. et al. Inhibition of influenza virus production in virus-infected mice by RNA interference. Proc. Natl. Acad. Sci. USA 101, 8676-8681 (2004).
26. Zhang, Y. et al. Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer. Clin. Cancer Res. 10, 3667-3677 (2004).
27. Hung, L. & Kumar, V. Specific inhibition of gene expression and transactivation functions of hepatitis B virus X protein and c-myc by small interfering RNAs. FEBS Lett. 560, 210-214 (2004).
28. Reich, S.J. et al. Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model. Mol. Vis. 9, 210-216 (2003).
29. Brody, J.R. et al. pp32 reduction induces differentiation of TSU-Pr1 cells. Am. J. Pathol. 164, 273-283 (2004).
30. Stewart, S.A. et al. Lentivirus-delivered stable gene silencing by RNAi in primary cells. RNA 9, 493-501 (2003).
31. Muthumani, K. et al. Novel engineered HIV-1 East African Clade-A gp160 plasmid construct induces strong humoral and cell-mediated immune responses in vivo. Virology 314, 134-146 (2003).