Improving the intracellular delivery and molecular efficacy of antisense oligonucleotides in chronic myeloid leukemia cells: A comparison of Streptolysin-O permeabilization, electroporation, and lipophilic conjugation

Blood

Volumn 91, Issue 12, 1998, Pages 4738-4746

  Spiller, David G ^b   Giles, Richard V ^b   Grzybowski, John ^b   Tidd, David M ^b   Clark, Richard E ^a  

^a ROYAL LIVERPOOL UNIVERSITY HOSPITAL   (United Kingdom)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA TOCOPHEROL; ANTISENSE OLIGODEOXYNUCLEOTIDE; CHOLESTEROL; FLUORESCEIN; LAURYL ALCOHOL; MACROGOL; PHOSPHOROTHIOIC ACID; POLOXAMER; STREPTOLYSIN O;

ARTICLE; CELL DIFFERENTIATION; CELL GROWTH; CELL MEMBRANE; CHIMERA; CHROMOSOME 22Q; CHROMOSOME 9Q; CHRONIC MYELOID LEUKEMIA; DRUG EFFICACY; DRUG UPTAKE; ELECTROPORATION; FLOW CYTOMETRY; FLUORESCENCE MICROSCOPY; HUMAN; LIPOPHILICITY; NORTHERN BLOTTING; PHENOTYPE; PHILADELPHIA 1 CHROMOSOME; PRIORITY JOURNAL; TARGET CELL;

EID: 0031811518     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v91.12.4738.412k31_4738_4746     Document Type: Article

References (45)

1. Groffen J, Stephenson JR, Heisterkamp N, de Klein A, Bartram CR, Grosveld G: Philadelphia chromosome breakpoints are clustered within a limited region, bcr, on chromosome 22. Cell 36:93, 1984
2. Szczylik C, Skorski T, Nicolaides NC, Manzella L, Malaguarnera L, Venturelli D, Gewirtz AM, Calabretta B: Selective inhibition of leukaemia cell proliferation by BCR-ABL antisense oligodeoxynucleotides. Science 253:562, 1991
3. O'Brien SG, Kirkland MA, Melo JV, Rao MH, Davidson RJ, McDonald C, Goldman JM: Antisense bcr-abl oligomers cause nonspecific inhibition of chronic myeloid leukaemia cell lines. Leukaemia 8:2156, 1994
4. Smetsers TFCM, Skorski T, van de Locht LTF, Wessels HMC, Pennings AHM, De Witte T, Calabretta B, Mensink EJBM: Antisense bcr-abl oligonucleotides induce apoptosis in the Philadelphia chromosome-positive cell line BV173. Leukemia 8:129, 1994
5. Vaerman JL, Lammineur C, Moureau P, Lewalle P, Deldime F, Blumenfeld M, Martial P: BCR-ABL antisense oligodeoxyribonucleotides suppress the growth of leukemic and normal hematopoietic cells by a sequence-specific but non-antisense mechanism. Blood 86:3891, 1995
6. Smetsers TFCM, van der Locht LTF, Pennings AHM, Wessels HMC, de Witte TM, Mensink EJBM: Phosphorothioate BCR-ABL antisense oligonucleotides induce cell death, but fail to reduce cellular BCR-ABL protein levels. Leukemia 9:118, 1995
7. Tidd DM: A potential role for antisense oligonucleotide analogues in the development of oncogene targeted cancer chemotherapy. Anticancer Res 10:1169, 1990
8. Gao WY, Storm C, Egan W, Cheng YC: Cellular pharmacology of phosphorothioate homooligodeoxynucleotides in human cells. Mol Pharmacol 43:45, 1993
9. Bergan R, Neckers L: How do antisense oligodeoxynucleotides inhibit the growth of chronic myelogenous leukemia cells? Blood 87:4019, 1996
10. Vaerman JL, Martiat P: How do antisense oligodeoxynucleotides inhibit the growth of chronic myelogenous leukemia cells? Blood 87:4020, 1996
11. Tari AM, Tucker SD, Deisseroth A, Lopez-B ere stein G: Liposomal delivery of methylphosphonate antisense oligonucleotides in chronic myelogenous leukemia. Blood 84:601, 1994
12. Tidd DM, Hawley P, Warenius HM, Gibson I: Evaluation of N-ras oncogene anti-sense, sense and nonsense sequence methylphosphonate oligonucleotide analogues. Anti-Cancer Drug Design 3:117, 1988
13. Giles RV, Spiller DG, Green JA, Clark RE, Tidd DM: Optimization of antisense oligodeoxynucleotide structure for targeting bcr-abl mRNA. Blood 86:744, 1995
14. Spiller DG, Tidd DM: The uptake kinetics of chimeric oligodeoxynucleotide analogues in human leukaemia MOLT-4 cells. Anti-Cancer Drug Design 7:115, 1992
15. Clark RE: Poor cellular uptake of antisense oligodeoxynucleotides: An obstacle to their use in chronic myeloid leukaemia. Leuk Lymphoma 19:189, 1995
16. Tidd DM: Specificity of antisense oligonucleotides. Perspect Drug Disco very Design 4:51, 1996
17. Tidd DM: Ribonuclease H-mediated antisense effects in intact human leukaemia cells. Biochem Soc Trans 24:619, 1996
18. Saison-Behmoaras T, Tocque B, Rey I, Chassignol M, Thuong NT, Helene C: Short modified antisense oligonucleotides directed against Ha-ras point mutation induce selective cleavage of the mRNA and inhibit T24 cells proliferation. EMBO J 10:1111, 1991
19. Boutorin AS, Gus'kova LV, Ivanova EM, Kobetz ND, Zarytova VF, Ryte AS, Yurchenko LV, Vlassov VV: Synthesis of alkylating oligonucleotide derivatives containing cholesterol or phenazium residues at their 3′-terminus and their interaction with DNA within mammalian cells. FEBS Lett 254:129, 1989
20. Bennett CF, Chiang MY, Chan H, Shoemaker JE, Mirabelli CK: Cationic lipids enhance cellular uptake and activity of phosphorothioate oligonucleotides. Mol Pharmacol 41:1023, 1992
21. Wagner RW: Gene inhibition using antisense oligodeoxynucleotides. Nature 372:333, 1994
22. Bergan R, Hakim F, Schwartz GN, Kyle E, Cepada R, Szabo JM, Fowler D, Gress R, Neckers L: Electroporation of synthetic oligodeoxynucleotides: A novel technique for ex vivo bone marrow purging. Blood 88:731, 1996
23. Giles RV, Spiller DG, Tidd DM: Detection of ribonuclease H generated mRNA fragments in human leukaemia cells following reversible membrane permeabilisation in the presence of antisense oligodeoxynucleotides. Antisense Res Dev 5:23, 1995
24. Spiller DG, Tidd DM: Nuclear delivery of antisense oligodeoxyribonucleotides through reversible permeabilisation of human leukaemia cells with Streptolysin O. Antisense Res Dev 5:13, 1995
25. Giles RV, Grzybowski J, Spiller DG, Tidd DM: Enhanced antisense effects resulting from an improved Streptolysin-O protocol for oligodeoxynucleotide delivery into human leukaemia cells. Nucleosides Nucleotides 16:1155, 1997
26. MacKellar C, Graham D, Will DW, Burgess S, Brown T: Synthesis and physical properties of anti-HIV antisense oligonucleotides bearing terminal lipophilic groups. Nucleic Acids Res 13:3411, 1992
27. Jaschke A, Furste JP, Nordhoff E, Hillenkamp F, Cech D, Erdmann VA: Synthesis and properties of oligodeoxyribonucleotidepolyethylene glycol conjugates. Nucleic Acids Res 22:4810, 1994
28. Will DW, Brown T: Synthesis of vitamin E containing oligonucleotides. Tetrahedron Lett 33:2729, 1992
29. Barry ELR, Gesek FA, Friedman PA: Introduction of antisense oligodeoxynucleotides into cells by permeabilisation with Streptolysin-O. Biotechniques 15:1016, 1993
30. Broughton CM, Spiller DG, Pender N, Komorovskaya M, Grzybowski J, Giles RV, Tidd DM, Clark RE: Preclinical studies of Streptolysin-O in enhancing antisense oligonucleotide uptake in harvests from chronic myeloid leukaemia patients. Leukemia 11:1435, 1997
31. Chomczynski P, Sacchi N: Single-step method of RNA isolation by acid guanidinium thiocyanate-phenol-chloroform extraction. Anal Biochem 162:156, 1987
32. Chomczynski P: Solubilization in formamide protects RNA from degradation. Nucleic Acids Res 20:3791, 1992
33. Giles RV, Spiller DG, Tidd DM: Chimaeric oligodeoxynucleotide analogues: Enhanced cell uptake of structures which direct ribonuclease H with high specificity. Anti-Cancer Drug Design 8:33, 1993
34. Höltke H-J, Kessler C: Non-radioactive labelling of RNA transcripts in vitro with the hapten digoxigenin (DIG); hybridization and ELISA-based detection. Nucleic Acids Res 18:5843, 1990
35. Giles RV, Tidd DM: Increased specificity for antisense oligodeoxynucleotide targeting of RNA cleavage by RNase H using chimeric methylphosphonodiester/phosphodiester structures. Nucleic Acids Res 20:763, 1992
36. Zhao Q, Song X, Waldschmidt T, Fisher E, Krieg AM: Oligonucleotide uptake in human hematopoietic cells is increased in leukemia and is related to cellular activation. Blood 88:1788, 1996
37. Spiller DG, Giles RV, Broughton CM, Grzybowski J, Ruddell CJ, Tidd DM, Clark RE: The influence of target protein half-life on the effectiveness of antisense oligonucleotide analogue mediated biological responses, (submitted)
38. Dhut S, Chaplin T, Young BD: BCR-ABL and BCR proteins; biochemical characterisation and localisation. Leukaemia 4:745, 1990
39. Mahon FX, Ripoche J, Pigeonnier V, Jazwiec B, Pigneux A, Moreau JF, Reiffers J: Inhibition of chronic myelogenous leukemia cells harboring a BCR-ABL B3A2 junction by antisense oligonucleotides targeted at the B2A2 junction. Exp Hematol 23:1606, 1995
40. Yaswen P, Stampfer M, Ghosh K, Cohen J: Effects of sequence of thioated oligonucleotides on cultured human mammary epithelial cells. Antisense Res Dev 3:67, 1993
41. Burgess TL, Fischer EF, Ross SL, Bready JV, Qian Y-X, Bayewitch LA, Cohen AM, Herrera CJ, Hu SS-F, Kramer TB, Lott FD, Martin FH, Pierce GF, Simonet L, Farrell CL: The antiproliferative activity of c-myb and c-myc antisense oligonucleotides in smooth muscle cells is caused by a non-antisense mechanism. Proc Natl Acad Sci USA 92:4051, 1995
42. Yamamato S, Yamamato T, Kataoka T, Kuramoto E, Yano O, Tokunaga T: Unique palindromic sequences in synthetic oligonucleotides are required to induce INF and augment IFN-mediated natural killer activity. J Immunol 148:4072, 1992
43. Gewirtz AM, Luger S, Sokol D, Goiwdin B, Stadtmauer E, Reccio A, Ratajczak MZ: Oligodeoxynucleotide therapeutics for human myelogenous leukaemia: Interim results. Blood 88:270a, 1996 (abstr, suppl 1)
44. de Fabritiis P, Amadori S, Petti MC, Mancini M, Montefusco E, Picardi A, Geiser T, Campbell K, Calabretta B, Mandelli F: In vitro purging with BCR-ABL antisense oligodeoxyribonucleotides does not prevent haematological reconstitution after autologous bone marrow transplantation. Leukemia 9:662, 1995
45. Clark RE, Grzybowski J, Pender N, Spiller DG, Broughton CM, Brammer C, Tidd DM: Transplantation of antisense oligonucleotide purged autografts in chronic myeloid leukaemia. Exp Hematol 25:781, 1997 (abstr)