Regulatable aptamers in medicine: Focus on antithrombotic strategies

Expert Opinion on Biological Therapy

Volumn 4, Issue 10, 2004, Pages 1641-1647

  Potti, Anil ^a   Rusconi, Christopher P ^a,b   Sullenger, Bruce A ^a   Orrel, Thomas L ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b Regado Biosciences Inc   (United States)

Author keywords

Aptamers; Effects; Efficacy; Reversible; SELEX

Indexed keywords

ACETYLSALICYLIC ACID; ANGIOGENESIS INHIBITOR; ANTICOAGULANT AGENT; ANTIDOTE; ANTIINFLAMMATORY AGENT; ANTIMITOTIC AGENT; ANTINEOPLASTIC AGENT; ANTISENSE OLIGONUCLEOTIDE; ANTITHROMBIN; APTAMER; APTAMER 9.3T; DNA; HEPARIN; IMMUNOMODULATING AGENT; MACROGOL; PEGAPTANIB; PROTAMINE; PROTEIN INHIBITOR; RNA DERIVATIVE; SINGLE STRANDED DNA; TICLOPIDINE; UNCLASSIFIED DRUG; VASCULOTROPIN INHIBITOR; WARFARIN;

BLEEDING; CLINICAL TRIAL; COMBINATORIAL LIBRARY; CONFORMATIONAL TRANSITION; DNA SEQUENCE; DRUG CLEARANCE; DRUG EFFECT; DRUG HALF LIFE; DRUG MECHANISM; DRUG STRUCTURE; DRUG USE; EVALUATION; HUMAN; MEDICAL TECHNOLOGY; MOLECULAR SIZE; NONHUMAN; PROTEIN BINDING; PROTEIN DEGRADATION; PROTEIN FUNCTION; PROTEIN ISOLATION; PROTEIN PURIFICATION; PROTEIN STABILITY; RETINA MACULA AGE RELATED DEGENERATION; REVIEW; SEQUENCE ANALYSIS; STRUCTURE ANALYSIS; SYSTEMATIC EVOLUTION OF LIGANDS BY EXPONENTIAL ENRICHMENT; THROMBOSIS PREVENTION;

ANTICOAGULANTS; ANTIDOTES; APTAMERS, NUCLEOTIDE; BIOLOGICAL AVAILABILITY; BLOOD COAGULATION; BLOOD COAGULATION FACTORS; CLINICAL TRIALS; FIBRINOLYTIC AGENTS; HUMANS; NUCLEIC ACID CONFORMATION; PROTEIN BINDING; SELEX APTAMER TECHNIQUE; THROMBOPHILIA; THROMBOSIS;

ANIMALIA;

EID: 5644302316     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.4.10.1641     Document Type: Review

References (27)

1. SULLENGER BA, GALLARDO HF, LINGERS GE, GILBOA E: Overexpression of TAR sequences renders cells resistant to human immunodeficiency virus replication. Cell (1990) 63(3):601-608.
2. MORISHITA R, GIBBONS GH, HORIUCHI M et al.: A gene therapy strategy using a transcription factor decoy of the E2F binding site inhibits smooth muscle proliferation in vivo. Proc. Natl. Acad. Sci. USA (1995) 92(13):5855-5859.
3. ELLINGTON AD, SZOSTAK JW: In vitro selection of RNA molecules that bind specific ligands. Nature (1990) 346:818-822.
4. TUERK C, GOLD L: Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase. Science (1990) 249:505-510.
5. OSBORNE SE, ELLINGTON AD: Nucleic acid selection and the challenge of combinatorial chemistry. Chem. Rev. (1997) 97(2):349-370.
6. BRODY EN, GOLD L: Aptamers as therapeutic and diagnostic agents. J. Biotechnol. (2000) 74(1):5-13.
7. WHITE RR, SULLENGER BA, RUSCONI CP: Developing aptamers into therapeutics. J. Clin. Invest. (2000) 106(8):929-934.
8. GOLD L, POLISKY B, UHLENBECK O, YARUS M: Diversity of oligonucleotide functions. Annu. Rev. Biochem. (1995) 64:763-797.
9. CHARLTON J, SENNELLO J, SMITH D: In vivo imaging of inflammation using an aptamer inhibitor of human neutrophil elastase. Chem. Biol. (1997) 4(11):809-816.
10. GOLDEN MC, COLLINS BD, WILLIS MC, KOCH TH: Diagnostic potential of PhotoSELEX-evolved ssDNA aptamers. J. Biotechnol. (2000) 81(3):167-178.
11. WHITE R, RUSCONI C, SCARDINO E et al.: Generation of species crossreactive aptamers using 'toggle' SELEX. Mol. Ther. (2001) 4(6):567-573.
12. WHITE RR, SHAN S, RUSCONI CP et al.: Inhibition of rat corneal angiogenesis by a nuclease-resistant RNA aptamer specific for angiopoietin-2. Proc. Natl. Acad. Sci. USA (2003) 100(9):5028-5033.
13. RUSCONI CP, YEH A, LYERLY HK, LAWSON JH, SULLENGER BA: Blocking the initiation of coagulation by RNA aptamers to Factor VIIa. Thromb. Haemost. (2000) 84:841-848.
14. RUSCONI CP, SCARDINO E, LAYZER J et al.: RNA aptamers as reversible antagonists of coagulation Factor IXa. Nature (2002) 419:90-94.
15. EYETECH STUDY GROUP: Preclinical and Phase IA clinical evaluation of an anti-VEGF pegylated aptamer (EYE001) for the treatment of exudative age-related macular degeneration. Retina (2002) 22(2):143-152.
16. TUCKER CE, CHEN LS, JUDKINS MB, FARMER JA, GILL SC, DROLET DW: Detection and plasma pharmacokinetics of an antivascular endothelial growth factor oligonucleotide-aptamer (NX1838) in rhesus monkeys. J. Chromatogr. B. Biomed. Sci. Appl. (1999) 732:203-212.
17. WATSON SR, CHANG YF, O'CONNELL D, WEIGAND L, RINGQUIST S, PARMA DH: Anti-L-selectin aptamers: binding characteristics, pharmacokinetic parameters, and activity against an intravascular target in vivo. Antisense Nucleic Acid Drug Dev. (2000) 10(2):63-75.
18. BOCK LC: Selection of single-stranded DNA molecules that bind and inhibit human thrombin. Nature (1992) 355:564-566.
19. RUCKMAN J, GREEN LS, BEESON J et al.: 2′-Fluoropyrimidine RNA-based aptamers to the 165-amino acid form of vascularendothelial growth factor (VEGF165). Inhibition of receptor binding and VEGF-induced vascular permeability through interactions requiring the exon 7-encoded domain. J. Biol. Chem. (1998) 273(32):20556-20567.
20. WU AM, CHEN W, RAUBITSCHEK A et al.: Tumour localization of anti-CEA single-chain Fvs: improved targeting by non-covalent dimers. Immunotechnology (1996) 2(1):21-36.
21. GOLD L: Oligonucleotides as research, diagnostic, and therapeutic agents. J. Biol. Chem. (1995) 270:13581-13584.
22. SANTULLI-MAROTTO S, NAIR SK, RUSCONI C, SULLENGER B, GILBOA E: Multivalent RNA aptamers that inhibit CTLA-4 and enhance tumour immunity. Cancer Res. (2003) 63(21):7483-7489.
23. DEANDA A JR, COUTRE SE, MOON MR et al.: Pilot study of the efficacy of a thrombin inhibitor for use during cardiopulmonary bypass. Ann. Thorac. Surg. (1994) 58(2):344-350.
24. LIU X, CAO G, DING H et al.: Screening of functional antidotes of RNA aptamers against bovine thrombin. FEBS Lett. (2004) 562(1-3):125-128.
25. FAMULOK M, MAYER G: Aptamers as tools in molecular biology and immunology. Curr. Top. Microbiol. Immunol. (1999) 243:123-136.
26. GOLD L, ALPER J: Keeping pace with genomics through combinatorial chemistry. Nat. Biotechnol. (1997) 15:297.
27. EYETECH STUDY GROUP: Antivascular endothelial growth factor therapy for subfoveal choroidal neovascularization secondary to age-related macular degeneration: Phase II study results. Ophthalmology (2003) 110(5):979-986.