Nucleic acid-based techniques for post-transcriptional regulation of molecular targets

Current Opinion in Nephrology and Hypertension

Volumn 12, Issue 4, 2003, Pages 415-421

  Jarad, George ^a   Simske, Jeffrey S ^a   Sedor, John R ^a   Schelling, Jeffrey R ^a,b  

^a METROHEALTH MEDICAL CENTER   (United States)

^b NONE   (United States)

Author keywords

Antisense oligonucleotide; Aptamer; Decoy RNA; Double stranded RNA; Kidney; Renal; Ribozyme; RNA interference; RNAi; SiRNA

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; APTAMER; DNA; GENE PRODUCT; MESSENGER RNA; NUCLEIC ACID; RIBOSOME RNA; RIBOZYME; TRANSFER RNA;

GENE FUNCTION; GENETIC CODE; GENETIC TRANSCRIPTION; HEREDITY; HUMAN; HYPERTENSION; KIDNEY DISEASE; PRIORITY JOURNAL; REVIEW; RNA PROCESSING; TRANSCRIPTION REGULATION;

ANIMALS; GENE SILENCING; GENE THERAPY; HUMANS; HYPERTENSION; KIDNEY DISEASES; OLIGONUCLEOTIDES, ANTISENSE; RNA; RNA PROCESSING, POST-TRANSCRIPTIONAL; RNA, CATALYTIC; RNA, SMALL INTERFERING;

EID: 0038159652     PISSN: 10624821     EISSN: None     Source Type: Journal    
DOI: 10.1097/00041552-200307000-00010     Document Type: Review

References (55)

1. Doudna JA, Cech TR. The chemical repertoire of natural ribozymes. Nature 2002; 418:222-228. This is a nice review from the laboratory that originally discovered ribozymes. This paper discusses different types of ribozymes and mechanisms of action.
2. Cech TR. Ribozymes, the first 20 years. Biochem Soc Trans 2001; 30:1162-1166.
3. Luyckx VA, Leclercq B, Dowland LK, Yu AS. Diet-dependent hypercalciuria in transgenic mice with reduced CLC5 chloride channel expression. Proc Natl Acad Sci U S A 1999; 96:12174-12179.
4. Reddy MA, Adler SG, Kim YS, et al. Interaction of MAPK and 12-lipoxygenase pathways in growth and matrix protein expression in mesangial cells. Am J Physiol Renal Physiol 2002; 283:F985-F994.
5. Rogers CS, Vanoye CG, Sullenger BA, George AL Jr. Functional repair of a mutant chloride channel using a trans-splicing ribozyme. J Clin Invest 2002; 110:1783-1789. This nicely written paper uses a ribozyme to restore a functional cell phenotype, thereby suggesting that trans-splicing ribozymes are feasible for therapy in single gene disorders.
6. Sullenger BA, Gilboa E. Emerging clinical applications of RNA. Nature 2002; 418:252-258. This excellent review describes the current state of clinical trials with ribozymes, antisense oligonucleotides, aptamers, siRNAs, as well as other RNA-based therapeutic strategies.
7. Usman N, Blatt LM. Nuclease-resistant synthetic ribozymes: developing a new class of therapeutics. J Clin Invest 2000; 106:1197-1202.
8. Opalinska JB, Gewirtz AM. Nucleic-acid therapeutics: basic principles and recent applications. Nat Rev Drug Discov 2002; 1:503-514. This good review discusses ribozymes, antisense oligonucleotides and RNAi. The paper also contains a table with comprehensive lists of clinical trials with these reagents.
9. Jansen B, Zangemeister-Wittke U. Antisense therapy for cancer - the time of truth. Lancet Oncol 2002; 3:672-683. This is a recent review devoted to antisense strategies for cancer therapeutics. The paper contains a good background section about mechanisms of antisense oligonucleotide action, a table describing ongoing clinical trials, and in-depth review about some of the targeted molecules.
10. Corey DR. Telomerase inhibition, oligonucleotides, and clinical trials. Oncogene 2002; 21:631-637.
11. Dean NM. Functional genomics and target validation approaches using antisense oligonucleotide technology. Curr Opin Biotechnol 2001; 12:622-625.
12. Tiscornia G, Singer O, Ikawa M, Verma IM. A general method for gene knockdown in mice by using lentiviral vectors expressing small interfering RNA. Proc Natl Acad Sci U S A 2003; 100:1844-1848.
13. Segawa H, Kaneko I, Takahashi A, et al. Growth-related renal type II Na/P, cotransporter. J Biol Chem 2002; 277:19665-19672.
14. + exchanger in M-1 mouse cortical collecting duct cells. Am J Physiol Renal Physiol 2002; 282:F649-F654.
15. Basnakian AG, Ueda N, Kaushal GP, et al. DNase I-like endonuclease in rat kidney cortex that is activated during ischemia/reperfusion injury. J Am Soc Nephrol 2002; 13:1000-1007.
16. Lin HL, Chen XM, Wang JZ, Yu ZH. Inhibition of apoptosis in rat mesangial cells by tissue inhibitor of metalloproteinase-1. Kidney Int 2002; 62:60-69.
17. 2+ channels in human glomerular mesangial cells. Am J Physiol Cell Physiol 2002; 283:C1390-C1398.
18. Martin J, Eynstone LV, Davies M, et al. The role of ADAM 15 in glomerular mesangial cell migration. J Biol Chem 2002; 277:33683-33689.
19. Senthil D, Ghosh CG, Bhandari BK, Kasinath BS. The type 2 vascular endothelial growth factor receptor recruits insulin receptor substrate-1 in its signalling pathway. Biochem J 2002; 368:49-56.
20. Wang X, Shaw S, Amiri F, et al. Inhibition of the Jak/STAT signaling pathway prevents the high glucose-induced increase in TGF-β and fibronectin synthesis in mesangial cells. Diabetes 2002; 51:3505-3509.
21. Seville RA, Nijjar S, Barnett MW, et al. Annexin IV (Xanx-4) has a functional role in the formation of pronephric tubules. Development 2002; 129:1693-1704.
22. Saulnier DM, Ghanbari H, Brandli AW. Essential function of Wnt-4 for tubulogenesis in the Xenopus pronephric kidney. Dev Biol 2002; 248:13-28.
23. Kanwar YS, Kumar A, Ota K, et al. Identification of developmentally regulated mesodermal-specific transcript in mouse embryonic metanephros. Am J Physiol Renal Physiol 2002; 282:F953-F965.
24. Ricker JL, Mata JE, Iversen PL, Gattone VH. c-myc antisense oligonucleotide treatment ameliorates murine ARPKD. Kidney Int 2002; 61 (Suppl 1):125-131.
25. Yokoi H, Mukoyama M, Sugawara A, et al. Role of connective tissue growth factor in fibronectin expression and tubulointerstitial fibrosis. Am J Physiol Renal Physiol 2002; 282:F933-F942.
26. 2B-adrenergic receptor messenger RNA in the central nervous system. Hypertension 2001; 38:1075-1080.
27. Pachori AS, Numan MT, Ferrario CM, et al. Blood pressure-independent attenuation of cardiac hypertrophy by AT(1)R-AS gene therapy. Hypertension 2002; 39:969-975.
28. Kagiyama S, Eguchi S, Frank GD, et al. Angiotensin II-induced cardiac hypertrophy and hypertension are attenuated by epidermal growth factor receptor antisense. Circulation 2002; 106:909-912.
29. Nasevicius A, Ekker SC. Effective targeted gene 'knockdown' in zebrafish. Nat Genet 2000; 26:216-220.
30. Sewell KL, Geary RS, Baker BF, et al. Phase I trial of ISIS 104838, a 2′-methoxyethyl modified antisense oligonucleotide targeting tumor necrosis factor-α. J Pharmacol Exp Ther 2002; 303:1334-1343.
31. Kutryk MJ, Foley DP, van den BM, et al. Local intracoronary administration of antisense oligonucleotide against c-myc for the prevention of in-stent restenosis: results of the randomized investigation by the Thoraxcenter of antisense DNA using local delivery and IVUS after coronary stenting (ITALICS) trial. J Am Coll Cardiol 2002; 39:281-287.
32. Maksymowych WP, Blackburn WD Jr, Tami JA, Shanahan WR Jr. A randomized, placebo controlled trial of an antisense oligodeoxynucleotide to intercellular adhesion molecule-1 in the treatment of severe rheumatoid arthritis. J Rheumatol 2002; 29:447-453.
33. Yacyshyn BR, Chey WY, Goff J, et al. Double blind, placebo controlled trial of the remission inducing and steroid sparing properties of an ICAM-1 antisense oligodeoxynucleotide, alicaforsen (ISIS 2302), in active steroid dependent Crohn's disease. Gut 2002; 51:30-36.
34. Ostendorf T, Kunter U, Gröne HJ, et al. Specific antagonism of PDGF prevents renal scarring in experimental glomerulonephritis. J Am Soc Nephrol 2001; 12:909-918.
35. 165 mediates glomerular endothelial repair. J Clin Invest 1999; 104:913-923.
36. Plasterk RH. RNA silencing: the genome's immune system. Science 2002; 296:1263-1265.
37. Volpe TA, Kidner C, Hall IM, et al. Regulation of heterochromatic silencing and histone H3 lysine-9 methylation by RNAi. Science 2002; 297:1833-1837.
38. Moohizuki K, Fine NA, Fujisawa T, Gorovsky MA. Analysis of a piwi-related gene implicates small RNAs in genome rearrangement in tetrahymena. Cell 2002; 110:689-699.
39. Taverna SD, Coyne RS, Allis CD. Methylation of histone h3 at lysine 9 targets programmed DNA elimination in tetrahymena. Cell 2002; 110:701-711.
40. Hannon GJ. RNA interference. Nature 2002; 418:244-251. This recent review focuses on mechanisms of naturally occurring siRNAs.
41. Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 411:494-498.
42. Jarad G, Wang B, Khan S, et al. Fas activation induces renal tubular epithelial cell β8 integrin expression and function in the absence of apoptosis. J Biol Chem 2002; 277:47826-47833.
43. Goruppi S, Bonventre JV, Kyriakis JM. Signaling pathways and late-onset gene induction associated with renal mesangial cell hypedrophy. EMBO J 2002; 21:5427-5436.
44. Miyagishi M, Taira K. U6 promoter-driven siRNAs with four uridine 3′ overhangs efficiently suppress targeted gene expression in mammalian cells. Nat Biotechnol 2002; 20:497-500.
45. Lee NS, Dohjima T, Bauer G, et al. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol 2002; 20:500-505.
46. Brummelkamp TR, Bernards R, Agami R. A system for stable expression of shod interfering RNAs in mammalian cells. Science 2002; 296:550-553.
47. Paddison PJ, Caudy AA, Bernstein E, et al. Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells. Genes Dev 2002; 16:948-958.
48. Yu JY, DeRuiter SL, Turner DL. RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells. Proc Natl Acad Sci U S A 2002; 99:6047-6052.
49. Paul CP, Good PD, Winer I, Engelke DR. Effective expression of small interfering RNA in human cells. Nat Biotechnol 2002; 20:505-508.
50. Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391:806-811.
51. Kamath RS, Fraser AG, Dong Y, et al. Systematic functional analysis of the Caenorhabditis elegans genome using RNAi. Nature 2003; 421:231-237.
52. McCaffrey AP, Meuse L, Pham TT, et al. Gene expression: RNA interference in adult mice. Nature 2002; 418:38-39.
53. Lewis DL, Hagstrom JE, Loomis AG, et al. Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. Nat Genet 2002; 32:107-108.
54. Hu WY, Myers CP, Kilzer JM, et al. Inhibition of retroviral pathogenesis by RNA interference. Curr Biol 2002; 12:1301-1311.
55. Xia H, Mao Q, Paulson HL, Davidson BL. siRNA-mediated gene silencing in vitro and in vivo. Nat Biotechnol 2002; 20:1006-1010.