An in vivo evaluation of amphiphilic, biodegradable peptide copolymers as siRNA delivery agents

International Journal of Pharmaceutics

Volumn 466, Issue 1-2, 2014, Pages 58-67

  Barrett, Stephanie E ^a   Abrams, Marc T ^a   Burke, Rob ^a   Carr, Brian A ^a   Crocker, Louis S ^a   Garbaccio, Robert M ^a   Howell, Bonnie J ^a   Kemp, Eric A ^a   Kowtoniuk, Robert A ^a   Latham, Andrew H ^a   Leander, Karen R ^a   Leone, Anthony M ^a   Patel, Mihir ^a   Pechenov, Sergey ^a   Pudvah, Nicole T ^a   Riley, Sean ^a   Sepp Lorenzino, Laura ^a   Walsh, Eileen S ^a   Williams, J Michael ^a   Colletti, Steven L ^a  

^a MERCK RESEARCH LABORATORIES   (United States)

Author keywords

Poly(peptides); Polymer conjugates; RNA delivery; siRNA

Indexed keywords

ALANINE AMINOTRANSFERASE; APOLIPOPROTEIN B; BIODEGRADABLE POLYPEPTIDE COPOLYMER; COPOLYMER; MESSENGER RNA; ORNITHINE; PHENYLALANINE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG; PEPTIDE; POLYMER;

ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BIODEGRADABILITY; CONJUGATE; CONTROLLED STUDY; DRUG EFFICACY; DRUG LIVER LEVEL; DRUG MEGADOSE; DRUG POTENCY; DRUG SCREENING; DRUG SYNTHESIS; FEMALE; GENE DELIVERY SYSTEM; IN VIVO STUDY; LIVER CELL; MOLECULAR WEIGHT; NONHUMAN; PRIORITY JOURNAL; RAT; STOICHIOMETRY; STRUCTURE ACTIVITY RELATION; ANIMAL; CHEMISTRY; GENETICS; LIVER; METABOLISM; SPRAGUE DAWLEY RAT;

ANIMALS; APOLIPOPROTEINS B; FEMALE; LIVER; MOLECULAR WEIGHT; ORNITHINE; PEPTIDES; PHENYLALANINE; POLYMERS; RATS, SPRAGUE-DAWLEY; RNA, MESSENGER; RNA, SMALL INTERFERING;

EID: 84896538657     PISSN: 03785173     EISSN: 18733476     Source Type: Journal    
DOI: 10.1016/j.ijpharm.2014.03.011     Document Type: Article

References (27)

1. T. Aliferis, H. Iatrou, and N. Hadjichristidis Living Polypeptides Biomacromolecules 5 2004 1653 1656 (Pubitemid 39335198)
2. R.D. Bagshaw, S.H. Pasternak, D.J. Mahuran, and J.W. Callahan Nicastrin is a resident lysosomal membrane protein Biochemical and Biophysical Research Communications 300 2003 615 618 (Pubitemid 36268879)
3. L.M. Berte, D.R. Callihan, J. Carlson, B.J. Charlton, C.D. Flaherty, M.H. Hopper, B. Kirkley, O. Ndimbie, J. Schiffgens, P.J. Stupca, N. Sudderth, J.I. Wilson, and S.M. Woodcock Laboratory Documents: Development and Control; Approved Guideline fifth ed. 2006 Clinical and Laboratory Standards Institute Wayne, Pennsylvania
4. Burrell, M. M.(Ed.), 1993. Enzymes of Molecular Biology (Methods in Molec. Biol.), pp. 271-276.
5. A.J. Convertine, D.S.W. Benoit, C.L. Duvall, A.S. Hoffman, and P.S. Stayton Development of a novel endosomolytic diblock copolymer for siRNA delivery Journal of Controlled Release 133 2009 221 229
6. T.J. Deming Living polymerization of α-amino acid-N- carboxyanhydrides Journal of Polymer Science Part A: Polymer Chemistry 38 2000 3011 3018
7. A. Fire, S. Xu, M.K. Montgomery, S.A. Kostas, S.E. Driver, and C.C. Mello Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans Nature 391 1998 806 811 (Pubitemid 28099681)
8. D. Fischer, Y. Li, B. Ahlemeyer, J. Krieglstein, and T. Kissel In vitro cytotoxicity testing of polycations: influence of polymer structure on cell viability and hemolysis Biomaterials 24 2003 1121 1131 (Pubitemid 36069121)
9. R.r. Gaspar, and R. Duncan Polymeric carriers: preclinical safety and the regulatory implications for design and development of polymer therapeutics Advanced Drug Delivery Reviews 61 2009 1220 1231
10. G.J.M. Habraken, K.H.R.M. Wilsens, C.E. Koning, and A. Heise Optimization of N-carboxyanhydride (NCA) polymerization by variation of reaction temperature and pressure Polymer Chemistry 2 2011 1322 1330
11. N. Hadjichristidis, H. Iatrou, M. Pitsikalis, and G. Sakellariou Synthesis of well-defined polypeptide-based materials via the ring-opening polymerization of α-amino acid N-carboxyanhydrides Chemical Reviews 109 2009 5528 5578
12. K. Itaka, N. Kanayama, N. Nishiyama, W.-D. Jang, Y. Yamasaki, K. Nakamura, H. Kawaguchi, and K. Kataoka Supramolecular nanocarrier of siRNA from PEG-based block catiomer carrying diamine side chain with distinctive pKa directed to enhance intracellular gene silencing Journal of the American Chemical Society 126 2004 13612 13613 (Pubitemid 39407229)
13. A. Kichler Gene transfer with modified polyethylenimines The Journal of Gene Medicine 6 2004 S3 S10
14. R. Kircheis, L. Wightman, and E. Wagner Design and gene delivery activity of modified polyethylenimines Advanced Drug Delivery Reviews 53 2001 341 358 (Pubitemid 34024914)
15. R.A. Kowtoniuk, T. Pei, C.M. DeAngelo, J.H. Waldman, E.N. Guidry, J.M. Williams, R.M. Garbaccio, and S.E. Barrett Optimization of an α-(amino acid)-N-carboxyanhydride polymerization using the high vacuum technique: examining the effects of monomer concentration, polymerization kinetics, polymer molecular weight, and monomer purity Journal of Polymer Science, Part A: Polymer Chemistry 2014 10.1002/pola.27132
16. S. Matsumoto, R.J. Christie, N. Nishiyama, K. Miyata, A. Ishii, M. Oba, H. Koyama, Y. Yamasaki, and K. Kataoka Environment-responsive block copolymer micelles with a disulfide cross-linked core for enhanced siRNA delivery Biomacromolecules 10 2008 119 127
17. M. Morille, C. Passirani, A. Vonarbourg, A. Clavreul, and J.-P. Benoit Progress in developing cationic vectors for non-viral systemic gene therapy against cancer Biomaterials 29 2008 3477 3496
18. D.L. Pickel, N. Politakos, A. Avgeropoulos, and J.M. Messman A mechanistic study of α-(amino acid)-N-carboxyanhydride polymerization: comparing initiation and termination events in high-vacuum and traditional polymerization techniques Macromolecules 42 2009 7781 7788
19. D.B. Rozema, D.L. Lewis, D.H. Wakefield, S.C. Wong, J.J. Klein, P.L. Roesch, S.L. Bertin, T.W. Reppen, Q. Chu, A.V. Blokhin, J.E. Hagstrom, and J.A. Wolff Dynamic polyconjugates for targeted in vivo delivery of siRNA to hepatocytes Proceedings of the National Academy of Sciences 104 2007 12982 12987 (Pubitemid 351737577)
20. M.G. Stanton, and S.L. Colletti Medicinal chemistry of siRNA delivery Journal of Medicinal Chemistry 53 2010 7887 7901
21. H. Sun, F. Meng, A.A. Dias, M. Hendriks, J. Feijen, and Z. Zhong α-Amino acid containing degradable polymers as functional biomaterials: rational design, synthetic pathway, and biomedical applications Biomacromolecules 12 2011 1937 1955
22. W. Vayaboury, O. Giani, H. Cottet, A. Deratani, and F. Schué Living polymerization of α-amino acid N-carboxyanhydrides (NCA) upon decreasing the reaction temperature Macromolecular Rapid Communications 25 2004 1221 1224
23. X.-L. Wang, S. Ramusovic, T. Nguyen, and Z.-R. Lu Novel polymerizable surfactants with pH-sensitive amphiphilicity and cell membrane disruption for efficient siRNA delivery Bioconjugate Chemistry 18 2007 2169 2177 (Pubitemid 350220022)
24. X.-L. Wang, T. Nguyen, D. Gillespie, R. Jensen, and Z.-R. Lu A multifunctional and reversibly polymerizable carrier for efficient siRNA delivery Biomaterials 29 2008 15 22 (Pubitemid 47599653)
25. A.L. Weber, and S.L. Miller Reasons for the occurrence of the twenty coded protein amino acids Journal of Molecular Evolution 17 1981 273 284 (Pubitemid 12235261)
26. K.A. Whitehead, R. Langer, and D.G. Anderson Knocking down barriers: advances in siRNA delivery Nature Reviews Drug Discovery 8 2009 129 138
27. T.S. Zimmermann, A.C.H. Lee, A. Akinc, B. Bramlage, D. Bumcrot, M.N. Fedoruk, J. Harborth, J.A. Heyes, L.B. Jeffs, M. John, A.D. Judge, K. Lam, K. McClintock, L.V. Nechev, L.R. Palmer, T. Racie, I. Röhl, S. Seiffert, S. Shanmugam, V. Sood, J. Soutschek, I. Toudjarska, A.J. Wheat, E. Yaworski, W. Zedalis, V. Koteliansky, M. Manoharan, H.-P. Vornlocher, and I. MacLachlan RNAi-mediated gene silencing in non-human primates Nature 441 2006 111 114