Receptor-targeted liposome-peptide-siRNA nanoparticles represent an efficient delivery system for MRTF silencing in conjunctival fibrosis

Scientific Reports

Volumn 6, Issue , 2016, Pages

  Yu Wai Man, Cynthia ^a   Tagalakis, Aristides D ^b   Manunta, Maria D ^b   Hart, Stephen L ^b   Khaw, Peng T ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

DRUG CARRIER; LIPOSOME; MACROGOL DERIVATIVE; NANOPARTICLE; PEPTIDE; SMALL INTERFERING RNA; TRANSCRIPTION FACTOR;

AMINO ACID SEQUENCE; ANTAGONISTS AND INHIBITORS; CELL CULTURE; CHEMISTRY; CYTOLOGY; EYE DISEASE; FIBROBLAST; FIBROSIS; GENETICS; HUMAN; METABOLISM; PATHOLOGY; REAL TIME POLYMERASE CHAIN REACTION; RNA INTERFERENCE; TRANSMISSION ELECTRON MICROSCOPY; WESTERN BLOTTING;

AMINO ACID SEQUENCE; BLOTTING, WESTERN; CELLS, CULTURED; DRUG CARRIERS; EYE DISEASES; FIBROBLASTS; FIBROSIS; HUMANS; LIPOSOMES; MICROSCOPY, ELECTRON, TRANSMISSION; NANOPARTICLES; PEPTIDES; POLYETHYLENE GLYCOLS; REAL-TIME POLYMERASE CHAIN REACTION; RNA INTERFERENCE; RNA, SMALL INTERFERING; TRANSCRIPTION FACTORS;

EID: 84959183074     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep21881     Document Type: Article

References (62)

1. Yin H., et al. Non-viral vectors for gene-based therapy. Nature Rev Genet 15, 541-555 (2014
2. Elbashir S. M., et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411, 494-498 (2001
3. Borel F., Kay M. A., & Mueller C. Recombinant AAV as a platform for translating the therapeutic potential of RNA interference. Mol Ther. 22, 692-701 (2014
4. Kang E. M., & Tisdale J. F. The leukemogenic risk of integrating retroviral vectors in hematopoietic stem cell gene therapy applications. Curr Hematol Rep 3, 274-281 (2004
5. Tagalakis A. D., He L., Saraiva L., Gustafsson K. T., & Hart S. L. Receptor-targeted liposome-peptide nanocomplexes for siRNA delivery. Biomaterials 32, 6302-6315 (2011
6. Tagalakis A. D., et al. Multifunctional, self-assembling anionic peptide-lipid nanocomplexes for targeted siRNA delivery. Biomaterials 35, 8406-8415 (2014
7. Kim B., et al. Inhibition of ocular angiogenesis by siRNA targeting vascular endothelial growth factor pathway genes -therapeutic strategy for herpetic stromal keratitis. Am J Pathol 165, 2177-2185 (2004
8. Jia R. B., et al. VEGF-targeted RNA interference suppresses angiogenesis, and tumor growth of retinoblastoma. Ophthalmic Res. 39, 108-115 (2007
9. Nakamura H., et al. RNA interference targeting transforming growth factor-beta type II receptor suppresses ocular inflammation, and fibrosis. Mol Vis 10, 703-711 (2004
10. Solano E. C., et al. Toxicological, and pharmacokinetic properties of QPI-1007, a chemically modified synthetic siRNA targeting caspase 2 mRNA, following intravitreal injection. Nucleic Acid Ther. 24, 258-266 (2014
11. Nguyen Q. D., et al. Evaluation of the siRNA PF-04523655 versus ranibizumab for the treatment of neovascular age-related macular degeneration (MONET Study). Ophthalmology 119, 1867-1873 (2012
12. Kaiser P. K., et al. RNAi-Based Treatment for Neovascular Age-Related Macular Degeneration by Sirna-027. Am J Ophthalmol 150, 33-39 (2010
13. Quigley H. A. Glaucoma. Lancet 377, 1367-1377 (2011
14. Small E. M., et al. Myocardin-related transcription factor-a controls myofibroblast activation, and fibrosis in response to myocardial infarction. Circ Res 107, 294-304 (2010
15. Luchsinger L. L., Patenaude C. A., Smith B. D., & Layne M. D. Myocardin-related transcription factor-A complexes activate type I collagen expression in lung fibroblasts. J Biol Chem 286, 44116-44125 (2011
16. Minami T., et al. Reciprocal expression of MRTF-A, and myocardin is crucial for pathological vascular remodelling in mice. EMBO J 31, 4428-4440 (2012
17. Yu-Wai-Man C., Treisman R. T., Bailly M., & Khaw P. T. The role of the MRTF-A/SRF pathway in Ocular fibrosis. Invest Ophthalmol Vis Sci. 55, 4560-4567 (2014
18. Manunta M. D., et al. Nebulisation of receptor-targeted nanocomplexes for gene delivery to the airway epithelium. PLoS ONE 6, e26768 (2011
19. Tagalakis A. D., et al. A receptor-targeted nanocomplex vector system optimized for respiratory gene transfer. Mol. Ther. 16, 907-915 (2008
20. Kenny G. D., et al. Multifunctional receptor-targeted nanocomplexes for magnetic resonance imaging, and transfection of tumours. Biomaterials 33, 7241-7250 (2012
21. Tagalakis A. D., et al. Integrin-targeted nanocomplexes for tumour specific delivery, and therapy by systemic administration. Biomaterials 32, 1370-1376 (2011
22. Irvine S. A., et al. Receptor-targeted nanocomplexes optimized for gene transfer to primary vascular cells, and explant cultures of rabbit aorta. Mol. Ther. 16, 508-515 (2008
23. Meng Q. H., et al. Inhibition of neointimal hyperplasia in a rabbit vein graft model following non-viral transfection with human iNOS cDNA. Gene Ther. 20, 979-986 (2013
24. Tagalakis A. D., Saraiva L., McCarthy D., Gustafsson K. T., & Hart S. L. Comparison of nanocomplexes with branched, and linear peptides for siRNA delivery. Biomacromolecules 14, 761-770 (2013
25. Tagalakis A. D., et al. PEGylation improves the receptor-mediated transfection efficiency of peptide-targeted, self-assembling, anionic nanocomplexes. J Control Release 174, 177-187 (2014
26. Tagalakis A. D., et al. A method for concentrating lipid peptide DNA, and siRNA nanocomplexes that retains their structure, and transfection efficiency. Int J Nanomedicine 10, 2673-2683 (2015
27. Dahlmann-Noor A. H., Martin-Martin B., Eastwood M., Khaw P. T., & Bailly M. Dynamic protrusive cell behaviour generates force, and drives early matrix contraction by fibroblasts. Exp Cell Res 313, 4158-4169 (2007
28. Daniels J. T., et al. Matrix metalloproteinase inhibition modulates fibroblast-mediated matrix contraction, and collagen production in vitro. Invest Ophthalmol Vis Sci. 44, 1104-1110 (2003
29. Jackson A. L., et al. Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol. 21, 635-637 (2003
30. Scacheri P. C., et al. Short interfering RNAs can induce unexpected, and divergent changes in the levels of untargeted proteins in mammalian cells. Proc Natl Acad Sci USA 101, 1892-1897 (2004
31. Caffrey D. R., et al. siRNA Off-Target Effects Can Be Reduced at Concentrations That Match Their Individual Potency. PLoS ONE. 6, e21503 (2011
32. Yu-Wai-Man C., & Khaw P. T. Developing novel anti-fibrotic therapeutics to modulate post-surgical wound healing in glaucoma: Big potential for small molecules. Expert Rev Ophthalmol. 10, 65-76 (2015
33. Du Z., Munye M. M., Tagalakis A. D., Manunta M. D., & Hart S. L. The role of the helper lipid on the DNA transfection efficiency of lipopolyplex formulations. Sci Rep 4, 7107 (2014
34. Munye M. M., et al. Role of liposome, and peptide in the synergistic enhancement of transfection with a lipopolyplex vector. Sci Rep 5, 9292 (2015
35. Wong T. T. L., Mead A. L., & Khaw P. T. Matrix Metalloproteinase Inhibition Modulates Postoperative Scarring after Experimental Glaucoma Filtration Surgery. Invest Ophthalmol Vis Sci. 44, 1097-1103 (2003
36. Cordeiro M. F., Gay J., & Khaw P. T. Human anti-transforming growth factor-beta2 antibody: a new glaucoma anti-scarring agent. Invest Ophthalmol Vis Sci 40, 2225-2234 (1999
37. Khaw P. T., Doyle J. W., Sherwood M. B., Smith M. F., & McGorray S. Effects of intraoperative 5-fluorouracil or mitomycin C on glaucoma filtration surgery in the rabbit. Ophthalmology 100, 367-372 (1993
38. Yorston D., & Khaw P. T. A randomised trial of the effect of intraoperative 5-FU on the outcome of trabeculectomy in east Africa. Br J Ophthalmol 85, 1028-1030 (2001
39. Lanigan L., Stürmer J., Baez K. A., Hitchings R. A., & Khaw P. T. Single intraoperative applications of 5-fluorouracil during filtration surgery: early results. Br J Ophthalmol 78, 33-37 (1994
40. Butler M. R., et al. Topical silver nanoparticles result in improved bleb function by increasing filtration, and reducing fibrosis in a rabbit model of filtration surgery. Invest Ophthalmol Vis Sci. 54, 4982-4990 (2013
41. Ye H., et al. Cationic nano-copolymers mediated IKKβ targeting siRNA to modulate wound healing in a monkey model of glaucoma filtration surgery. Mol Vis. 16, 2502-2510 (2010
42. Bhattacharjee S., et al. Surface charge-specific cytotoxicity, and cellular uptake of tri-block copolymer nanoparticles. Nanotoxicology. 7, 71-84 (2013
43. Pan Y., et al. Size-dependent cytotoxicity of gold nanoparticles. Small. 3, 1941-1949 (2007
44. Carlson C., et al. Unique cellular interaction of silver nanoparticles: size-dependent generation of reactive oxygen species. J Phys Chem B. 112, 13608-13619 (2008
45. Yu K. O., et al. Toxicity of amorphous silica nanoparticles in mouse keratinocytes. J Nanopart Res. 11, 15-24 (2009
46. Prabhu B. M., Ali S. F., Murdock R. C., Hussain S. M., & Srivatsan M. Copper nanoparticles exert size, and concentration dependent toxicity on somatosensory neurons of rat. Nanotoxicology. 4, 150-160 (2010
47. Win K. Y., & Feng S. S. Effects of particle size, and surface coating on cellular uptake of polymeric nanoparticles for oral delivery of anticancer drugs. Biomaterials. 26, 2713-2722 (2005
48. Johnston H. J., et al. Evaluating the uptake, and intracellular fate of polystyrene nanoparticles by primary, and hepatocyte cell lines in vitro. Toxicol Appl Pharmacol. 242, 66-78 (2010
49. Foged C., Brodin B., Frokjaer S., & Sundblad A. Particle size, and surface charge affect particle uptake by human dendritic cells in an in vitro model. Int J Pharm. 298, 315-322 (2005
50. Oh W.-K., et al. Cellular uptake, cytotoxicity, and innate immune response of silica-titania hollow nanoparticles based on size, and surface functionality. ACS Nano. 4, 5301-5313 (2010
51. Zhang S. L., Li J., Lykotrafitis G., Bao G., & Suresh S. Size-dependent endocytosis of nanoparticles. Adv Mater. 21, 419-424 (2009
52. Whitehead K. A., Langer R., & Anderson D. G. Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov 8, 129-138 (2009
53. Hart S. L., Collins L., Gustafsson K., & Fabre J. W. Integrin-mediated transfection with peptides containing arginine-glycineaspartic acid domains. Gene Therapy 4, 1225-1230 (1997
54. Masur S. K., Cheung J. K. H., & Antohi S. Identification of Integrins in Cultured Corneal Fibroblasts, and in Isolated Keratocytes. Invest Ophthalmol Vis Sci 34, 2690-2698 (1993
55. Writer M. J., et al. Targeted gene delivery to human airway epithelial cells with synthetic vectors incorporating novel targeting peptides selected by phage display. J Drug Target 12, 185-193 (2004
56. Huang F. W., et al. PEGylated PEI-based biodegradable polymers as non-viral gene vectors. Acta Biomater. 6, 4285-4295 (2010
57. Mishra S., Webster P., & Davis M. E. PEGylation significantly affects cellular uptake, and intracellular trafficking of non-viral gene delivery particles. Eur J Cell Biol. 83, 97-111 (2004
58. Jere D., et al. Poly (amino ester) composed of poly (ethylene glycol), and aminosilane prepared by combinatorial chemistry as a gene carrier. Pharm Res. 25, 875-885 (2008
59. Corsi K., Chellat F., Yahia L., & Fernandes J. C. Mesenchymal stem cells, MG63, and HEK293 transfection using chitosan-DNA nanoparticles. Biomaterials 24, 1255-1264 (2003
60. Ladewig K., Niebert M., Xu Z. P., Gray P. P., & Lu G. Q. Efficient siRNA delivery to mammalian cells using layered double hydroxide nanoparticles. Biomaterials 31, 1821-1829 (2010
61. Lee K. J., et al. In vivo quantitative study of sized-dependent transport, and toxicity of single silver nanoparticles using zebrafish embryos. Chem Res Toxicol. 25, 1029-1046 (2012
62. Tan Y. F., et al. Layer-by-layer nanoparticles as an efficient siRNA delivery vehicle for SPARC silencing. Small 10, 1790-1798 (2014