RNA interference for therapy in the vascular endothelium

Microvascular Research

Volumn 80, Issue 2, 2010, Pages 286-293

  Kaufmann, Jörg ^a   Ahrens, Katharina ^a   Santel, Ansgar ^a  

^a Silence Therapeutics GmbH   (Germany)

Author keywords

Angiogenesis; Atu027; Endothelial dysfunction; RNAi therapeutics

Indexed keywords

ATU 027; ATUFECT 01; ATUPLEX; CAVEOLIN; CD7 ANTIGEN; COPPER ZINC SUPEROXIDE DISMUTASE; DRUG CARRIER; ENDOTHELIAL LEUKOCYTE ADHESION MOLECULE 1; LIPOPLEX; MACROGOL; NANOPARTICLE; PHOSPHATIDYLINOSITOL 3 KINASE; PHOSPHATIDYLINOSITOL 3,4,5 TRISPHOSPHATE 3 PHOSPHATASE; SMALL INTERFERING RNA; TOLL LIKE RECEPTOR 3; TUMOR NECROSIS FACTOR ALPHA; UNCLASSIFIED DRUG; VASCULOTROPIN RECEPTOR 2;

ANTIANGIOGENIC GENE THERAPY; ANTIINFLAMMATORY ACTIVITY; ANTISENSE THERAPY; BASEMENT MEMBRANE; BLOOD BRAIN BARRIER; BLOOD VESSEL PERMEABILITY; CANCER GENE THERAPY; CANCER INHIBITION; CELL PROLIFERATION; DIABETIC MACULAR EDEMA; ENDOTHELIUM CELL; GENE EXPRESSION; GENE FUNCTION; GENE SILENCING; GENE TARGETING; GENE TECHNOLOGY; GLYCOCALYX; HUMAN; INTERNALIZATION; LEUKOCYTE ACTIVATION; LIPOSOMAL GENE DELIVERY SYSTEM; MACROPHAGE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PHAGOCYTOSIS; PRIORITY JOURNAL; PROSTATE CANCER; RETINA MACULA AGE RELATED DEGENERATION; REVIEW; RHEUMATOID ARTHRITIS; RNA INTERFERENCE; T LYMPHOCYTE; TIGHT JUNCTION; TUMOR VASCULARIZATION; VASCULAR DISEASE; VASCULAR ENDOTHELIUM;

EID: 77955419350     PISSN: 00262862     EISSN: 10959319     Source Type: Journal    
DOI: 10.1016/j.mvr.2010.02.002     Document Type: Review

References (56)

1. Aird W.C. The role of the endothelium in severe sepsis and multiple organ dysfunction syndrome. Blood 2003, 101:3765-3777.
2. Aird W.C. Spatial and temporal dynamics of the endothelium. J. Thromb. Haemost. 2005, 3:1392-1406.
3. Aird W.C. Phenotypic heterogeneity of the endothelium: I. Structure, function, and mechanisms. Circ. Res. 2007, 100:158-173.
4. Aird W.C. Phenotypic heterogeneity of the endothelium: II. Representative vascular beds. Circ. Res. 2007, 100:174-190.
5. Akira S., Uematsu S., Takeuchi O. Pathogen recognition and innate immunity. Cell 2006, 124:783-801.
6. Aleku M., Fisch G., Mopert K., Keil O., Arnold W., Kaufmann J., Santel A. Intracellular localization of lipoplexed siRNA in vascular endothelial cells of different mouse tissues. Microvasc. Res. 2008, 76:31-41.
7. Aleku M., Schulz P., Keil O., Santel A., Schaeper U., Dieckhoff B., Janke O., Endruschat J., Durieux B., Roder N., Loffler K., Lange C., Fechtner M., Mopert K., Fisch G., Dames S., Arnold W., Jochims K., Giese K., Wiedenmann B., Scholz A., Kaufmann J. Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression. Cancer Res. 2008, 68:9788-9798.
8. Asgeirsdottir S.A., Talman E.G., de Graaf I.A., Kamps J.A., Satchell S.C., Mathieson P.W., Ruiters M.H., Molema G. Targeted transfection increases siRNA uptake and gene silencing of primary endothelial cells in vitro-a quantitative study. J. Control Release 2010, 141:241-251.
9. Braet F., Wisse E. Structural and functional aspects of liver sinusoidal endothelial cell fenestrae: a review. Comp. Hepatol. 2002, 1:1-17.
10. Campbell M., Kiang A.S., Kenna P.F., Kerskens C., Blau C., O'Dwyer L., Tivnan A., Kelly J.A., Brankin B., Farrar G.J., Humphries P. RNAi-mediated reversible opening of the blood-brain barrier. J. Gene Med. 2008, 10:930-947.
11. Cho W.G., Albuquerque R.J., Kleinman M.E., Tarallo V., Greco A., Nozaki M., Green M.G., Baffi J.Z., Ambati B.K., De Falco M., Alexander J.S., Brunetti A., De Falco S., Ambati J. Small interfering RNA-induced TLR3 activation inhibits blood and lymphatic vessel growth. Proc. Natl. Acad. Sci. U. S. A. 2009, 106:7137-7142.
12. Cines D.B., Pollak E.S., Buck C.A., Loscalzo J., Zimmerman G.A., McEver R.P., Pober J.S., Wick T.M., Konkle B.A., Schwartz B.S., Barnathan E.S., McCrae K.R., Hug B.A., Schmidt A.M., Stern D.M. Endothelial cells in physiology and in the pathophysiology of vascular disorders. Blood 1998, 91:3527-3561.
13. Courties G., Presumey J., Duroux-Richard I., Jorgensen C., Apparailly F. RNA interference-based gene therapy for successful treatment of rheumatoid arthritis. Expert. Opin. Biol. Ther. 2009, 9:535-538.
14. Czauderna F., Fechtner M., Dames S., Aygun H., Klippel A., Pronk G.J., Giese K., Kaufmann J. Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells. Nucleic Acids Res. 2003, 31:2705-2716.
15. Czauderna F., Santel A., Hinz M., Fechtner M., Durieux B., Fisch G., Leenders F., Arnold W., Giese K., Klippel A., Kaufmann J. Inducible shRNA expression for application in a prostate cancer mouse model. Nucleic Acids Res. 2003, e127:31.
16. Durr E., Yu J., Krasinska K.M., Carver L.A., Yates J.R., Testa J.E., Oh P., Schnitzer J.E. Direct proteomic mapping of the lung microvascular endothelial cell surface in vivo and in cell culture. Nat. Biotechnol. 2004, 22:985-992.
17. Elbashir S.M., Harborth J., Lendeckel W., Yalcin A., Weber K., Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001, 411:494-498.
18. Folkman J. Angiogenesis: an organizing principle for drug discovery?. Nat. Rev., Drug Discov. 2007, 6:273-286.
19. Gasparini G., Longo R., Toi M., Ferrara N. Angiogenic inhibitors: a new therapeutic strategy in oncology. Nat. Clin. Pract. Oncol. 2005, 2:562-577.
20. Hadj-Slimane R., Lepelletier Y., Lopez N., Garbay C., Raynaud F. Short interfering RNA (siRNA), a novel therapeutic tool acting on angiogenesis. Biochimie 2007, 89:1234-1244.
21. Hobson B., Denekamp J. Endothelial proliferation in tumours and normal tissues: continuous labelling studies. Br. J. Cancer 1984, 49:405-413.
22. Howard K.A., Paludan S.R., Behlke M.A., Besenbacher F., Deleuran B., Kjems J. Chitosan/siRNA nanoparticle-mediated TNF-alpha knockdown in peritoneal macrophages for anti-inflammatory treatment in a murine arthritis model. Mol. Ther. 2009, 17:162-168.
23. Kalluri R. Basement membranes: structure, assembly and role in tumour angiogenesis. Nat. Rev., Cancer 2003, 3:422-433.
24. Kawakami S., Hashida M. Targeted delivery systems of small interfering RNA by systemic administration. Drug Metab. Pharmacokinet. 2007, 22:142-151.
25. Khoury M., Escriou V., Courties G., Galy A., Yao R., Largeau C., Scherman D., Jorgensen C., Apparailly F. Efficient suppression of murine arthritis by combined anticytokine small interfering RNA lipoplexes. Arthritis Rheum. 2008, 58:2356-2367.
26. Kim V.N., Han J., Siomi M.C. Biogenesis of small RNAs in animals. Nat. Rev., Mol. Cell. Biol. 2009, 10:126-139.
27. Kleinman M.E., Yamada K., Takeda A., Chandrasekaran V., Nozaki M., Baffi J.Z., Albuquerque R.J., Yamasaki S., Itaya M., Pan Y., Appukuttan B., Gibbs D., Yang Z., Kariko K., Ambati B.K., Wilgus T.A., DiPietro L.A., Sakurai E., Zhang K., Smith J.R., Taylor E.W., Ambati J. Sequence- and target-independent angiogenesis suppression by siRNA via TLR3. Nature 2008, 452:591-597.
28. Koh W., Mahan R.D., Davis G.E. Cdc42- and Rac1-mediated endothelial lumen formation requires Pak2, Pak4 and Par3, and PKC-dependent signaling. J. Cell. Sci. 2008, 121:989-1001.
29. Koh W., Sachidanandam K., Stratman A.N., Sacharidou A., Mayo A.M., Murphy E.A., Cheresh D.A., Davis G.E. Formation of endothelial lumens requires a coordinated PKC{epsilon}-, Src-, Pak- and Raf-kinase-dependent signaling cascade downstream of Cdc42 activation. J. Cell. Sci. 2009, 122:1812-1822.
30. Kumar P., Wu H., McBride J.L., Jung K.E., Kim M.H., Davidson B.L., Lee S.K., Shankar P., Manjunath N. Transvascular delivery of small interfering RNA to the central nervous system. Nature 2007, 448:39-43.
31. Kumar P., Ban H.S., Kim S.S., Wu H., Pearson T., Greiner D.L., Laouar A., Yao J., Haridas V., Habiro K., Yang Y.G., Jeong J.H., Lee K.Y., Kim Y.H., Kim S.W., Peipp M., Fey G.H., Manjunath N., Shultz L.D., Lee S.K., Shankar P. T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 2008, 134:577-586.
32. Leenders F., Mopert K., Schmiedeknecht A., Santel A., Czauderna F., Aleku M., Penschuck S., Dames S., Sternberger M., Rohl T., Wellmann A., Arnold W., Giese K., Kaufmann J., Klippel A. PKN3 is required for malignant prostate cell growth downstream of activated PI 3-kinase. EMBO J. 2004, 23:3303-3313.
33. Lu P.Y., Xie F.Y., Woodle M.C. Modulation of angiogenesis with siRNA inhibitors for novel therapeutics. Trends Mol. Med. 2005, 11:104-113.
34. Maniatis N.A., Orfanos S.E. The endothelium in acute lung injury/acute respiratory distress syndrome. Curr. Opin. Crit. Care 2008, 14:22-30.
35. Mehta D., Malik A.B. Signaling mechanisms regulating endothelial permeability. Physiol. Rev. 2006, 86:279-367.
36. Miyawaki-Shimizu K., Predescu D., Shimizu J., Broman M., Predescu S., Malik A.B. siRNA-induced caveolin-1 knockdown in mice increases lung vascular permeability via the junctional pathway. Am. J. Physiol. Lung Cell Mol. Physiol. 2006, 290:L405-413.
37. Möpert K., Hajek P., Frank S., Chen C., Kaufmann J., Santel A. Loss of Drp1 function alters OPA1 processing and changes mitochondrial membrane organization. Exp. Cell. Res. 2009, 315:2165-2180.
38. Morille M., Passirani C., Vonarbourg A., Clavreul A., Benoit J.P. Progress in developing cationic vectors for non-viral systemic gene therapy against cancer. Biomaterials 2008, 29:3477-3496.
39. Opalinska J.B., Gewirtz A.M. Nucleic-acid therapeutics: basic principles and recent applications. Nat. Rev., Drug Discov. 2002, 1:503-514.
40. Opitz B., Hippenstiel S., Eitel J., Suttorp N. Extra- and intracellular innate immune recognition in endothelial cells. Thromb. Haemost. 2007, 98:319-326.
41. Peer D., Park E.J., Morishita Y., Carman C.V., Shimaoka M. Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target. Science 2008, 319:627-630.
42. Pober J.S., Min W., Bradley J.R. Mechanisms of endothelial dysfunction, injury, and death. Annu. Rev. Pathol. 2009, 4:71-95.
43. Pries A.R., Kuebler W.M. Normal endothelium. Handb. Exp. Pharmacol. 2006, 1-40.
44. Pries A.R., Secomb T.W., Gaehtgens P. The endothelial surface layer. Pflugers Arch. 2000, 440:653-666.
45. Reich S.J., Fosnot J., Kuroki A., Tang W., Yang X., Maguire A.M., Bennett J., Tolentino M.J. Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model. Mol. Vis. 2003, 9:210-216.
46. Santel A., Aleku M., Keil O., Endruschat J., Esche V., Durieux B., Loffler K., Fechtner M., Rohl T., Fisch G., Dames S., Arnold W., Giese K., Klippel A., Kaufmann J. RNA interference in the mouse vascular endothelium by systemic administration of siRNA-lipoplexes for cancer therapy. Gene Ther. 2006, 13:1360-1370.
47. Santel A., Aleku M., Keil O., Endruschat J., Esche V., Fisch G., Dames S., Loffler K., Fechtner M., Arnold W., Giese K., Klippel A., Kaufmann J. A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium. Gene Ther. 2006, 13:1222-1234.
48. Schiffelers R.M., Ansari A., Xu J., Zhou Q., Tang Q., Storm G., Molema G., Lu P.Y., Scaria P.V., Woodle M.C. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic Acids Res. 2004, e149:32.
49. Seaman S., Stevens J., Yang M.Y., Logsdon D., Graff-Cherry C., St Croix B. Genes that distinguish physiological and pathological angiogenesis. Cancer Cell. 2007, 11:539-554.
50. Thurston G., McLean J.W., Rizen M., Baluk P., Haskell A., Murphy T.J., Hanahan D., McDonald D.M. Cationic liposomes target angiogenic endothelial cells in tumors and chronic inflammation in mice. J. Clin. Invest. 1998, 101:1401-1413.
51. Tseng Y.C., Mozumdar S., Huang L. Lipid-based systemic delivery of siRNA. Adv. Drug Deliv. Rev. 2009, 61:721-731.
52. Vandenbroucke E., Mehta D., Minshall R., Malik A.B. Regulation of endothelial junctional permeability. Ann. N.Y. Acad. Sci. 2008, 1123:134-145.
53. Venturini L., Eder M., Scherr M. RNA-mediated gene silencing in hematopoietic cells. J. Biomed. Biotechnol. 2006, 2006:87340.
54. Whitehead K.A., Langer R., Anderson D.G. Knocking down barriers: advances in siRNA delivery. Nat. Rev., Drug Discov. 2009, 8:129-138.
55. Winderlich M., Keller L., Cagna G., Broermann A., Kamenyeva O., Kiefer F., Deutsch U., Nottebaum A.F., Vestweber D. VE-PTP controls blood vessel development by balancing Tie-2 activity. J. Cell. Biol. 2009, 185:657-671.
56. Wisse E. An electron microscopic study of the fenestrated endothelial lining of rat liver sinusoids. J. Ultrastruct. Res. 1970, 31:125-150.