A nonpolycationic fully proteinaceous multiagent system for potent targeted delivery of siRNA

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages e162-

  Liu, David V ^a   Yang, Nicole J ^a   Wittrup, K Dane ^a,b  

^a Massachusetts Institute of Technology   (United States)

^b MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

Author keywords

Cholesterol dependent cytolysin; DsRBD; Protein based siRNA delivery

Indexed keywords

CHOLESTEROL; CYTOLYSIN; DOUBLE STRANDED RNA; EPIDERMAL GROWTH FACTOR RECEPTOR; HYBRID PROTEIN; SMALL INTERFERING RNA;

ARTICLE; CYTOPLASM; CYTOTOXICITY; ENDOSOME; ENZYME ACTIVITY; ENZYME MECHANISM; GENE DELIVERY SYSTEM; GENE SILENCING; HUMAN; HUMAN CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; RNA INTERFERENCE;

EID: 84901228277     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.14     Document Type: Article

References (31)

1. Jarvis L (2009). Delivering the promise. Chem Eng News 87:18-27.
2. Alexis, F, Pridgen, E, Molnar, LK and Farokhzad, OC (2008). Factors affecting the clearance and biodistribution of polymeric nanoparticles. Mol Pharm 5: 505-515.
3. Schmidt, MM and Wittrup, KD (2009). A modeling analysis of the effects of molecular size and binding affinity on tumor targeting. Mol Cancer Ther 8: 2861-2871.
4. Kumar, P, Ban, HS, Kim, SS, Wu, H, Pearson, T, Greiner, DL et al. (2008). T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 134: 577-586.
5. Song, E, Zhu, P, Lee, SK, Chowdhury, D, Kussman, S, Dykxhoorn, DM et al. (2005). Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat Biotechnol 23: 709-717.
6. Peer, D, Zhu, P, Carman, CV, Lieberman, J and Shimaoka, M (2007). Selective gene silencing in activated leukocytes by targeting siRNAs to the integrin lymphocyte functionassociated antigen-1. Proc Natl Acad Sci USA 104: 4095-4100.
7. Winkler, J, Martin-Killias, P, Plückthun, A and Zangemeister-Wittke, U (2009). EpCAMtargeted delivery of nanocomplexed siRNA to tumor cells with designed ankyrin repeat proteins. Mol Cancer Ther 8: 2674-2683.
8. Niesner, U, Halin, C, Lozzi, L, Günthert, M, Neri, P, Wunderli-Allenspach, H et al. (2002). Quantitation of the tumor-targeting properties of antibody fragments conjugated to cellpermeating HIV-1 TAT peptides. Bioconjug Chem 13: 729-736.
9. Lee, HJ and Pardridge, WM (2001). Pharmacokinetics and delivery of tat and tat-protein conjugates to tissues in vivo. Bioconjug Chem 12: 995-999.
10. Bevilacqua, PC and Cech, TR (1996). Minor-groove recognition of double-stranded RNA by the double-stranded RNA-binding domain from the RNA-activated protein kinase PKR. Biochemistry 35: 9983-9994.
11. Hackel, BJ, Ackerman, ME, Howland, SW and Wittrup, KD (2010). Stability and CDR composition biases enrich binder functionality landscapes. J Mol Biol 401: 84-96.
12. Eguchi, A, Meade, BR, Chang, YC, Fredrickson, CT, Willert, K, Puri, N et al. (2009). Efficient siRNA delivery into primary cells by a peptide transduction domain-dsRNA binding domain fusion protein. Nat Biotechnol 27: 567-571.
13. Kim, J, Lee, SH, Choe, J and Park, TG (2009). Intracellular small interfering RNA delivery using genetically engineered double-stranded RNA binding protein domain. J Gene Med 11: 804-812.
14. Geoghegan, JC, Gilmore, BL and Davidson, BL (2012). Gene silencing mediated by siRNA-binding fusion proteins is attenuated by double-stranded RNA-binding domain structure. Mol Ther Nucleic Acids 1: e53.
15. Rosado, CJ, Kondos, S, Bull, TE, Kuiper, MJ, Law, RH, Buckle, AM et al. (2008). The MACPF/CDC family of pore-forming toxins. Cell Microbiol 10: 1765-1774.
16. Spangler, JB, Manzari, MT, Rosalia, EK, Chen, TF and Wittrup, KD (2012). Triepitopic antibodies inhibit cetuximab-resistant BRAF-and KRAS mutant tumors via EGFR signal repression. J Mol Biol 28:532-544.
17. Fu, Q and Yuan, YA (2013). Structural insights into RISC assembly facilitated by dsRNAbinding domains of human RNA helicase A (DHX9). Nucleic Acids Res 41: 3457-3470.
18. Nanduri, S, Carpick, BW, Yang, Y, Williams, BR and Qin, J (1998). Structure of the doublestranded RNA-binding domain of the protein kinase PKR reveals the molecular basis of its dsRNA-mediated activation. EMBO J 17: 5458-5465.
19. Pirie, CM, Liu, DV and Wittrup, KD (2013). Targeted cytolysins synergistically potentiate cytoplasmic delivery of gelonin immunotoxin. Mol Cancer Ther 12: 1774-1782.
20. Schuerch, DW, Wilson-Kubalek, EM and Tweten, RK (2005). Molecular basis of listeriolysin O pH dependence. Proc Natl Acad Sci USA 102: 12537-12542.
21. Pirie, CM, Hackel, BJ, Rosenblum, MG and Wittrup, KD (2010). Convergent potency of internalized gelonin immunotoxins across varied cell lines, antigens, and targeting moieties. J Biol Chem 286: 4165-4172.
22. Liang, W and Lam, JKW (2012). Endosomal escape pathways for non-viral nucleic acid delivery systems. In: Ceresa, B (ed.). Molecular Regulation of Endocytosis. Intech: Rijeka, pp. 429-456.
23. Dominska, M and Dykxhoorn, DM (2010). Breaking down the barriers: siRNA delivery and endosome escape. J Cell Sci 123: 1183-1189.
24. Barnes, DW (1982). Epidermal growth factor inhibits growth of A431 human epidermoid carcinoma in serum-free cell culture. J Cell Biol 93: 1-4.
25. Perera, RM, Zoncu, R, Johns, TG, Pypaert, M, Lee, FT, Mellman, I et al. (2007). Internalization, intracellular trafficking, and biodistribution of monoclonal antibody 806: a novel anti-epidermal growth factor receptor antibody. Neoplasia 9: 1099-1110.
26. de Bono, JS and Rowinsky, EK (2002). The ErbB receptor family: a therapeutic target for cancer. Trends Mol Med 8: S19-S26.
27. Wakeling, AE (2002). Epidermal growth factor receptor tyrosine kinase inhibitors. Curr Opin Pharmacol 2: 382-387.
28. Dunstone, MA and Tweten, RK (2012). Packing a punch: the mechanism of pore formation by cholesterol dependent cytolysins and membrane attack complex/perforin-like proteins. Curr Opin Struct Biol 22: 342-349.
29. Geiser, M, Cèbe, R, Drewello, D and Schmitz, R (2001). Integration of PCR fragments at any specific site within cloning vectors without the use of restriction enzymes and DNA ligase. Biotechniques 1: 88-92.
30. Spanggord, RJ and Beal, PA (2000). Site-specific modification and RNA crosslinking of the RNA-binding domain of PKR. Nucleic Acids Res 28: 1899-1905.
31. Liu, DV, Maier, LM, Hafler, DA and Wittrup, KD (2009). Engineered interleukin-2 antagonists for the inhibition of regulatory T cells. J Immunother 32: 887-894