Nanosized multifunctional polyplexes for receptor-mediated SiRNA delivery

ACS Nano

Volumn 6, Issue 6, 2012, Pages 5198-5208

  Dohmen, Christian ^a   Edinger, Daniel ^a   Fröhlich, Thomas ^a   Schreiner, Laura ^a   Lächelt, Ulrich ^a   Troiber, Christina ^a   Rädler, Joachim ^a   Hadwiger, Philipp ^b   Vornlocher, Hans Peter ^b   Wagner, Ernst ^a  

^a UNIVERSITY OF MUNICH   (Germany)

^b AXOLABS GMBH   (Germany)

Author keywords

endosomal escape; folate targeting; lytic peptide; polyplex; RNAi

Indexed keywords

ENDOSOMAL ESCAPE; FOLATE TARGETING; LYTIC PEPTIDES; POLYPLEXES; RNAI;

AMIDES; AMINO ACIDS; GENES; GENETIC ENGINEERING; OLIGOMERS; PEPTIDES; POLYETHYLENE GLYCOLS; SYNTHESIS (CHEMICAL); TISSUE;

RNA;

NANOCAPSULE; SMALL INTERFERING RNA;

ANIMAL; ARTICLE; CHEMISTRY; EXPERIMENTAL NEOPLASM; GENE SILENCING; GENETICS; MATERIALS TESTING; MOUSE; TREATMENT OUTCOME;

ANIMALS; GENE SILENCING; MATERIALS TESTING; MICE; NANOCAPSULES; NEOPLASMS, EXPERIMENTAL; RNA, SMALL INTERFERING; TREATMENT OUTCOME;

EID: 84862847986     PISSN: 19360851     EISSN: 1936086X     Source Type: Journal    
DOI: 10.1021/nn300960m     Document Type: Article

References (36)

1. Gao, K.; Huang, L. Nonviral Methods for SiRNA Delivery Mol. Pharm. 2009, 6, 651-658
2. Wang, J.; Lu, Z.; Wientjes, M. G.; Au, J. L. Delivery of SiRNA Therapeutics: Barriers and Carriers AAPS J. 2010, 12, 492-503
3. Alexis, F.; Pridgen, E.; Molnar, L. K.; Farokhzad, O. C. Factors Affecting the Clearance and Biodistribution of Polymeric Nanoparticles Mol. Pharm. 2008, 5, 505-515
4. Kwok, A.; Hart, S. L. Comparative Structural and Functional Studies of Nanoparticle Formulations for DNA and SiRNA Delivery Nanomedicine 2011, 7, 210-219
5. Meade, B. R.; Dowdy, S. F. The Road to Therapeutic RNA Interference (RNAi): Tackling the 800 Pound Sirna Delivery Gorilla Discovery Med. 2009, 8, 253-256
6. Xing, J.; Deng, L.; Guo, S.; Dong, A.; Liang, X. J. Polycationic Nanoparticles as Nonviral Vectors Employed for Gene Therapy in Vivo Mini-Rev. Med. Chem. 2010, 10, 126-137
7. Wagner, E. Polymers for SiRNA Delivery: Inspired by Viruses to Be Targeted, Dynamic, and Precise. Acc. Chem. Res. 2012, not supplied.
8. Andaloussi, S. E.; Lehto, T.; Mager, I.; Rosenthal-Aizman, K.; Oprea, I. I.; Simonson, O. E.; Sork, H.; Ezzat, K.; Copolovici, D. M.; Kurrikoff, K. et al. Design of a Peptide-Based Vector, Pepfect6, for Efficient Delivery of SiRNA in Cell Culture and Systemically in Vivo Nucleic Acids Res. 2011, 39, 3972-3987
9. Matsumoto, S.; Christie, R. J.; Nishiyama, N.; Miyata, K.; Ishii, A.; Oba, M.; Koyama, H.; Yamasaki, Y.; Kataoka, K. Environment-Responsive Block Copolymer Micelles with a Disulfide Cross-Linked Core for Enhanced SiRNA Delivery Biomacromolecules 2009, 10, 119-127
10. Rozema, D. B.; Lewis, D. L.; Wakefield, D. H.; Wong, S. C.; Klein, J. J.; Roesch, P. L.; Bertin, S. L.; Reppen, T. W.; Chu, Q.; Blokhin, A. V. et al. Dynamic Polyconjugates for Targeted in Vivo Delivery of SiRNA to Hepatocytes Proc. Natl. Acad. Sci. U. S. A. 2007, 104, 12982-12987
11. Creusat, G.; Thomann, J. S.; Maglott, A.; Pons, B.; Dontenwill, M.; Guerin, E.; Frisch, B.; Zuber, G. Pyridylthiourea-Grafted Polyethylenimine Offers an Effective Assistance to SiRNA-Mediated Gene Silencing in Vitro and in Vivo J. Controlled Release 2012, 157, 418-426
12. Mao, S.; Neu, M.; Germershaus, O.; Merkel, O.; Sitterberg, J.; Bakowsky, U.; Kissel, T. Influence of Polyethylene Glycol Chain Length on the Physicochemical and Biological Properties of Poly(Ethylene Imine)-Graft- Poly(Ethylene Glycol) Block Copolymer/SiRNA Polyplexes Bioconjugate Chem. 2006, 17, 1209-1218
13. Malek, A.; Czubayko, F.; Aigner, A. Peg Grafting of Polyethylenimine (PEI) Exerts Different Effects on DNA Transfection and SiRNA-Induced Gene Targeting Efficacy J. Drug Targeting 2008, 16, 124-139
14. Taratula, O.; Garbuzenko, O. B.; Kirkpatrick, P.; Pandya, I.; Savla, R.; Pozharov, V. P.; He, H.; Minko, T. Surface-Engineered Targeted PPI Dendrimer for Efficient Intracellular and Intratumoral SiRNA Delivery J. Controlled Release 2009, 140, 284-293
15. Walker, G. F.; Fella, C.; Pelisek, J.; Fahrmeir, J.; Boeckle, S.; Ogris, M.; Wagner, E. Toward Synthetic Viruses: Endosomal pH-Triggered Deshielding of Targeted Polyplexes Greatly Enhances Gene Transfer in Vitro and in Vivo Mol. Ther. 2005, 11, 418-425
16. Meyer, M.; Philipp, A.; Oskuee, R.; Schmidt, C.; Wagner, E. Breathing Life into Polycations: Functionalization with pH-Responsive Endosomolytic Peptides and Polyethylene Glycol Enables SiRNA Delivery J. Am. Chem. Soc. 2008, 130, 3272-3273
17. Ikeda, Y.; Taira, K. Ligand-Targeted Delivery of Therapeutic SiRNA Pharm. Res. 2006, 23, 1631-1640
18. Plank, C.; Oberhauser, B.; Mechtler, K.; Koch, C.; Wagner, E. The Influence of Endosome-Disruptive Peptides on Gene Transfer Using Synthetic Virus-Like Gene Transfer Systems J. Biol. Chem. 1994, 269, 12918-12924
19. Wagner, E.; Plank, C.; Zatloukal, K.; Cotten, M.; Birnstiel, M. L. Influenza Virus Hemagglutinin HA-2 N-Terminal Fusogenic Peptides Augment Gene Transfer by Transferrin-Polylysine-DNA Complexes: Toward a Synthetic Virus-Like Gene-Transfer Vehicle Proc. Natl. Acad. Sci. U. S. A. 1992, 89, 7934-7938
20. Meyer, M.; Dohmen, C.; Philipp, A.; Kiener, D.; Maiwald, G.; Scheu, C.; Ogris, M.; Wagner, E. Synthesis and Biological Evaluation of a Bioresponsive and Endosomolytic SiRNA-Polymer Conjugate Mol. Pharm. 2009, 6, 752-762
21. Zuber, G.; Zammut-Italiano, L.; Dauty, E.; Behr, J. P. Targeted Gene Delivery to Cancer Cells: Directed Assembly of Nanometric DNA Particles Coated with Folic Acid Angew. Chem., Int. Ed. 2003, 42, 2666-2669
22. Erbacher, P.; Remy, J. S.; Behr, J. P. Gene Transfer with Synthetic Virus-Like Particles Via the Integrin-Mediated Endocytosis Pathway Gene Ther. 1999, 6, 138-145
23. DeRouchey, J.; Walker, G. F.; Wagner, E.; Radler, J. O. Decorated Rods: A "Bottom-Up" Self-Assembly of Monomolecular DNA Complexes J. Phys. Chem. B 2006, 110, 4548-4554
24. DeRouchey, J.; Schmidt, C.; Walker, G. F.; Koch, C.; Plank, C.; Wagner, E.; Radler, J. O. Monomolecular Assembly of SiRNA and Poly(Ethylene Glycol)-Peptide Copolymers Biomacromolecules 2008, 9, 724-732
25. Akinc, A.; Goldberg, M.; Qin, J.; Dorkin, J. R.; Gamba-Vitalo, C.; Maier, M.; Jayaprakash, K. N.; Jayaraman, M.; Rajeev, K. G.; Manoharan, M. et al. Development of Lipidoid-SiRNA Formulations for Systemic Delivery to the Liver Mol. Ther. 2009, 17, 872-879
26. Hartmann, L.; Hafele, S.; Peschka-Suss, R.; Antonietti, M.; Borner, H. G. Tailor-Made Poly(Amidoamine)s for Controlled Complexation and Condensation of DNA Chemistry 2008, 14, 2025-2033
27. Schaffert, D.; Troiber, C.; Salcher, E. E.; Frohlich, T.; Martin, I.; Badgujar, N.; Dohmen, C.; Edinger, D.; Klager, R.; Maiwald, G. et al. Solid-Phase Synthesis of Sequence-Defined T-, I-, and U-Shape Polymers for pDNA and SiRNA Delivery Angew. Chem., Int. Ed. 2011, 50, 8986-8989
28. Schaffert, D.; Badgujar, N.; Wagner, E. Novel Fmoc-Polyamino Acids for Solid-Phase Synthesis of Defined Polyamidoamines Org. Lett. 2011, 13, 1586-1589
29. Fröhlich, T.; Edinger, D.; Klager, R.; Troiber, C.; Salcher, E.; Badgujar, N.; Martin, I.; Schaffert, D.; Cengizeroglu, A.; Hadwiger, P. et al. Structure-Activity Relationships of SiRNA Carriers Based on Sequence-Defined Oligo (Ethane Amino) Amides J. Controlled Release 2012, 160, 532-541
30. Wang, S.; Lee, R. J.; Mathias, C. J.; Green, M. A.; Low, P. S. Synthesis, Purification, and Tumor Cell Uptake of 67Ga-Deferoxamine - Folate, a Potential Radiopharmaceutical for Tumor Imaging Bioconjugate Chem. 1996, 7, 56-62
31. Boeckle, S.; von Gersdorff, K.; van der Piepen, S.; Culmsee, C.; Wagner, E.; Ogris, M. Purification of Polyethylenimine Polyplexes Highlights the Role of Free Polycations in Gene Transfer J. Gene Med. 2004, 6, 1102-1111
32. Plank, C.; Mechtler, K.; Szoka, F. C., Jr.; Wagner, E. Activation of the Complement System by Synthetic DNA Complexes: A Potential Barrier for Intravenous Gene Delivery Hum. Gene Ther. 1996, 7, 1437-1446
33. Hamad, I.; Al-Hanbali, O.; Hunter, A. C.; Rutt, K. J.; Andresen, T. L.; Moghimi, S. M. Distinct Polymer Architecture Mediates Switching of Complement Activation Pathways at the Nanosphere-Serum Interface: Implications for Stealth Nanoparticle Engineering ACS Nano 2010, 4, 6629-6638
34. Kamen, B. A.; Capdevila, A. Receptor-Mediated Folate Accumulation Is Regulated by the Cellular Folate Content Proc. Natl. Acad. Sci. U. S. A. 1986, 83, 5983-5987
35. Xia, W.; Low, P. S. Folate-Targeted Therapies for Cancer J. Med. Chem. 2010, 53, 6811-6824
36. Judge, A. D.; Robbins, M.; Tavakoli, I.; Levi, J.; Hu, L.; Fronda, A.; Ambegia, E.; McClintock, K.; MacLachlan, I. Confirming the RNAi-Mediated Mechanism of Action of SiRNA-Based Cancer Therapeutics in Mice J. Clin. Invest. 2009, 119, 661-673