Endosomolysis by masking of a membrane-active agent (EMMA) for cytoplasmic release of macromolecules

Bioconjugate Chemistry

Volumn 14, Issue 1, 2003, Pages 51-57

  Rozema, David B ^a   Ekena, Kirk ^a   Lewis, David L ^a   Loomis, Aaron G ^a   Wolff, Jon A ^b  

^a Mirus Bio Corporation   (United States)

^b UNIVERSITY OF WISCONSIN SCHOOL OF MEDICINE AND PUBLIC HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMINO ACIDS; CYTOLOGY; NUCLEIC ACIDS; OLIGONUCLEOTIDES; PEPTIDES;

ACIDIC CONDITIONS; ACTIVE AGENTS; AMIDE BOND; CATIONIC MEMBRANES; CYTOPLASMIC RELEASE; ENDOSOMES; MELITTIN; MEMBRANE DISRUPTION; MEMBRANE-ACTIVE PEPTIDES; NEUTRAL PH;

MEMBRANES;

AMIDE; MALEIC ANHYDRIDE; MELITTIN; MORPHOLINE DERIVATIVE; OLIGONUCLEOTIDE;

ACYLATION; ARTICLE; CHEMICAL BOND; CYTOTOXICITY; DRUG DELIVERY SYSTEM; ENDOSOME; HUMAN; HUMAN CELL; MACROMOLECULE; MEMBRANE DAMAGE; PH;

EMMA;

EID: 0037257707     PISSN: 10431802     EISSN: None     Source Type: Journal    
DOI: 10.1021/bc0255945     Document Type: Article

References (45)

1. Brown, M. D., Schatslein, A. G., and Uchegbu, I. F. (2001) Gene delivery with synthetic (non viral) carriers. Int. J. Pharm. 229, 1-21.
2. Lloyd, J. B. (2000) Lysosome membrane permeability: implications for drug Del.ery. Adv. Drug Del. Rev. 41, 189-200.
3. Nishikawa, M., and Huang, L. (2001) Nonviral vectors in the new millenium: delivery Barrier in Gene Transfer. Human Gene Ther. 12, 861-870.
4. Drummond, D. C., Zignani, M., and Leroux, J.-C. (2000) Current status of pH-sensitive liposomes in drug delivery. Prog. Lipid Res. 39, 409-460.
5. Eckert, D. M., and Kim, P. S. (2001) Mechanisms of viral membrane fusion and its inhibition. Annu. Rev. Biochem. 70, 777-810.
6. Nemerow, G. R., and Stewart, P. L. (1999) Role of alpha v integrins in adenovirus cell entry and gene delivery. Microbiol. Mol. Biol. Rev. 63, 725-734.
7. Asokan, A., and Cho, M. J. (2002) Exploitation of intracellular pH gradients in the cellular delivery of macromolecules. J. Pharm. Sci. 91, 903-913.
8. Carrasco, L. (1994) Entry of animal viruses and macromolecules into cells. FEBS Lett. 350, 151-4.
9. Clague, M. J. (1998) Molecular aspects of the endocytic pathway. Biochem. J. 336, 271-282.
10. Gruenberg, J. (2001) The endocytic pathway: A mosaic of domains. Nat. Rev.: Mol. Cell. Biol. 2, 721-730.
11. Mukherjee, S., Ghosh, R. N., and Maxfield, F. R. (1997) Endocytosis. Physiol. Rev. 77, 759-803.
12. Ciftci, K., and Levy, R. J. (2001) Enhanced plasmid DNA transfection with lysosomotropic agents in cultured fibroblasts. Int. J. Pharm., 218, 81-92.
13. Kichler, A., Leborgne, C., Coeytaux, E., and Danos, O. (2001) Polyethylenimine-mediated gene Delivery: a mechanistic study. J. Gene Med. 3, 135-144.
14. Klemm, A. R., Young, D., and Lloyd, J. B. (1998) Effects of polyethyleneimine on endocytosis and lysosome stability. Biochem. Pharm. 56, 41-46.
15. Zuber, G., et al. (2001) Towards synthetic viruses. Adv. Drug Del. Rev. 52, 245-53.
16. Budker, V., et al. (1996) pH-sensitive, cationic liposomes: a new synthetic virus-like vector. Nat. Biotech. 14, 760-764.
17. Sitaram, N., and Nagaraj, R. (1999) Interaction of antimicrobial peptides with biological and model membranes: structural and charge requirements for activity. Biochim. Biophys. Acta 1462, 29-54.
18. Cheung, C. Y., Murthy, N., Stayton, P. S., and Hoffman, A. S. (2001) A pH-sensitive polymer that enhances cationic lipid-mediated gene transfer. Bioconjugate Chem. 12, 906-910.
19. Plank, C., Zauner, W., and Wagner, E. (1998) Application of membrane-active peptides for drug and gene delivery across cellular membranes. Adv. Drug Del. Rev. 34, 21-35.
20. Guo, X., and Szoka, F. C. (2001) Steric Stabilization of Fusogenic Liposomes by a Low-pH Sensitive PEG-Diortho Ester-Lipid Conjugate. Bioconjugate Chem. 12, 291-300.
21. Reddy, J. A., and Low, P. S. (2000) Enhanced folate receptor mediated gene therapy using a novel pH-sensitive lipid formulation. J. Controlled Release 64, 27-37.
22. Blattler, W. A., Kuenzi, B. S., Lambert, J. M., and Senter, P. D. (1985) New Heterobifunctional Protein Cross-Linking Reagent That Forms an Acid-Labile Link. Biochemistry 24, 1517-1524.
23. Naganawa, A., Ichikawa, Y., and Isobe, M. T. (1994) Synthetic Studies on Tautomycin Synthesis of 2,3-Disubstituted Maleic Anhydride Segment. Tetrahedron 50, 8969.
24. Trubetskoy, V. S., et al. (1999) Quantitative assessment of DNA condensation. Anal. Biochem. 267, 309-13.
25. Tang, M. X., and Szoka, F. C. (1997) The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther. 4, 823-32.
26. Lackey, C. A., et al. (1999) Hemolytic Activity of pH-Responsive Polymer-Streptavidin Bioconjugates. Bioconjugate Chem. 10, 401.
27. Wolff, J. A., et al. (1990) Direct gene transfer into mouse muscle in vivo. Science 247, 1465-8.
28. Kirby, A. J., and Lancaster, P. W. (1972) Structure and Efficiency in Intramolecular and Enzymic Catalysis. Catalysis of Amide Hydrolysis by the Carboxy-group of Substituted Maleamic Acids. J. Chem. Soc., Perkin Trans. 2 1206-1214.
29. Nieto, M. A., and Palacian, E. (1983) Effects of temperature and pH on the regeneration of the amino groups of ovalbumin after modification with citraconic and dimethylmaleic anhydrides. Biochim. Biophys Acta 749, 204-210.
30. Kirby, A. J. (1980) Effective Molarities for Intramolecular Reactions. Adv. Phys. Org. Chem. 17, 183-278.
31. Dempsey, C. E. (1990) The actions of melittin on membranes. Biochim. Biophys. Acta 1031, 143-161.
32. Blondelle, S. E., and Houghten, R. A. (1991) Hemolytic and antimicrobial activities of the twenty-four individual omission analogues of melittin. Biochemistry 30, 4671-8.
33. Yoshimura, A. (1985) Adenovirus -induced leakage of coendocytosed macromolecules into the cytosol. Cell Struct. Funct. 10, 391-404.
34. Budker, V., et al. (1997) Protein/amphipathic polyamine complexes enable highly efficient transfection with minimal toxicity. Biotechniques 23, 139, 142-147.
35. Heasman, J., Kofron, M., and Wylie, C. (2000) Beta-catenin signaling activity dissected in the early Xenopus embryo: a novel antisense approach. Dev. Biol. 222, 124-34.
36. Nasevicius, A., and Ekker, S. C. (2000) Effective targeted gene 'knockdown' in zebrafish. Nat. Genetics 26, 216-20.
37. Ghosh, C., and Iversen, P. L. (2000) Intracellular delivery strategies for antisense phosphorodiamidate morpholino oligomers. Antisense Nucleic Acid Drug Dev. 10, 263-74.
38. Giles, R. V., Spiller, D. G., Clark, R. E., and Tidd, D. M. (1999) Antisense morpholino oligonucleotide analogue induces missplicing of C-myc mRNA. Antisense Nucleic Acid Drug Dev. 9, 213-20.
39. Kang, S. H., Cho, M. J., and Kole, R. (1998) Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. Biochemistry 37, 6235-9.
40. Summerton, J., and Weller, D. (1997) Morpholino antisense oligomers: design, preparation, and properties. Antisense Nucleic Acid Drug Dev. 7, 187-95.
41. Akhtar, S., Basu, S., Wickstrom, E., and Juliano, R. L. (1991) Interactions of antisense DNA oligonucleotide analogues with phospholipid membranes (liposomes). Nucleic Acids Res. 19, 5551-9.
42. Akhtar, S., et al. (2000) The delivery of antisense therapeutics. Adv. Drug Del. Rev. 44, 3-21.
43. Audouy, S., and Hoekstra, D. (2001) Cationic lipid-mediated transfection in vitro and in vivo. Mol. Membr. Biol. 18, 129-43.
44. Hope, M. J., Mui, B., Ansell, S., and Ahkong, Q. F. (1998) Cationic lipids, phosphatidylethanolamine and the intracellular delivery of polymeric, nucleic acid-based drugs (Review). Mol. Mem. Biol. 15, 1-14.
45. Robaczewska, M., et al. (2001) Inhibition of hepadnaviral replication by polyethylenimine-based intravenous delivery of antisense phosphodiester oligodeoxynucleotides to the liver. Gene Ther. 8, 874-881.