Aptamer-based therapeutics of the past, present and future: From the perspective of eye-related diseases

Drug Discovery Today

Volumn 19, Issue 9, 2014, Pages 1309-1321

  Kanwar, Jagat R ^a   Shankaranarayanan, Jayanth Suryanarayanan ^a   Gurudevan, Sneha ^a   Kanwar, Rupinder K ^a  

^a DEAKIN UNIVERSITY   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

ALDEHYDE REDUCTASE; APTAMER; ARC 1905; ARC 81; ARC 81119; AX 102; BETA ADRENERGIC RECEPTOR; BEVASIRANIB; CASPASE 2; FIBROBLAST GROWTH FACTOR; FIBRONECTIN; FOMIVIRSEN; IMMUNOGLOBULIN ENHANCER BINDING PROTEIN; INDUCIBLE NITRIC OXIDE SYNTHASE; MORPHOLINO OLIGONUCLEOTIDE; NUCLEIC ACID; PEGAPTANIB; PEROXISOME PROLIFERATOR ACTIVATED RECEPTOR; PF 655; PLATELET DERIVED GROWTH FACTOR; PROSTAGLANDIN SYNTHASE; PROTEIN KINASE C; QPI 1007; RANIBIZUMAB; RUBOXISTAURIN; SMALL INTERFERING RNA; SYL 040012; TRANSFORMING GROWTH FACTOR BETA; UNCLASSIFIED DRUG; VASCULOTROPIN; VASCULOTROPIN RECEPTOR 1;

AGE RELATED MACULAR DEGENERATION; CHEMICAL MODIFICATION; DIABETIC RETINOPATHY; DRUG PROTEIN BINDING; EYE DISEASE; EYE INFECTION; GLAUCOMA; HUMAN; NONHUMAN; RETINITIS; REVIEW; ANIMAL; CHEMISTRY; DRUG DESIGN; EYE DISEASES; MOLECULARLY TARGETED THERAPY; PATHOPHYSIOLOGY;

ANIMALS; APTAMERS, NUCLEOTIDE; DRUG DESIGN; EYE DISEASES; HUMANS; MOLECULAR TARGETED THERAPY; NUCLEIC ACIDS;

EID: 84907816763     PISSN: 13596446     EISSN: 18785832     Source Type: Journal    
DOI: 10.1016/j.drudis.2014.02.009     Document Type: Review

References (126)

1. N. Congdon Causes and prevalence of visual impairment among adults in the United States Arch. Ophthalmol. 122 2004 477 485
2. R.E. Marquis, and J.T. Whitson Management of glaucoma: focus on pharmacological therapy Drugs Aging 22 2005 1 21
3. J.J. Servat, and C.R. Bernardino Effects of common topical antiglaucoma medications on the ocular surface, eyelids and periorbital tissue Drugs Aging 28 2011 267 282
4. J.C. Burnett, and J.J. Rossi RNA-based therapeutics: current progress and future prospects Chem. Biol. 19 2012 60 71
5. Z. Ahmed Ocular neuroprotection by siRNA targeting caspase-2 Cell Death Dis. 2 2011 e173
6. N.M. Bressler Age-related macular degeneration Survey Ophthalmol. 32 1988 375 413
7. R.D. Jager Age-related macular degeneration N. Eng. J. Med. 358 2008 2606 2617
8. N.S. Dejneka Ocular biodistribution of bevasiranib following a single intravitreal injection to rabbit eyes Mol. Vis. 14 2008 997 1005
9. J. Shen Suppression of ocular neovascularization with siRNA targeting VEGF receptor 1 Gene Ther. 13 2006 225 234
10. D.U. Lee Retina expression and cross-species validation of gene silencing by PF-655, a small interfering RNA against RTP801 for the treatment of ocular disease J. Ocul. Pharmacol. Ther. 28 2012 222 230
11. H. Qian, and H. Ripps Neurovascular interaction and the pathophysiology of diabetic retinopathy Exp. Diabetes Res. 2011 2011 693426
12. Pañeda, C. et al. (2012) Recent advances in ocular nucleic acid-based therapies: the silent era. doi: 10.5772/48454
13. J. Li Subconjunctival antisense oligonucleotides targeting TNF-alpha influence immunopathology and viral replication in murine HSV-1 retinitis Graefes. Arch. Clin. Exp. Ophthalmol. 246 2008 1265 1273
14. M. Moerdyk-Schauwecker Inhibition of HSV-1 ocular infection with morpholino oligomers targeting ICP0 and ICP27 Antiviral Res. 84 2009 131 141
15. S. Wasmuth Topical treatment with antisense oligonucleotides targeting tumor necrosis factor-alpha in herpetic stromal keratitis Invest. Ophthalmol. Vis. Sci. 44 2003 5228 5234
16. S. Wasmuth Topical antisense-oligonucleotides targeting IFN-gamma mRNA improve incidence and severity of herpetic stromal keratitis by cytokine specific and sequence unspecific effects Graefes. Arch. Clin. Exp. Ophthalmol. 246 2008 443 451
17. R.M. Orr Technology evaluation: fomivirsen, Isis Pharmaceuticals Inc/CIBA vision Curr. Opin. Mol. Ther. 3 2001 288 294
18. A.P. Adamis Increased vascular endothelial growth factor levels in the vitreous of eyes with proliferative diabetic retinopathy Am. J. Ophthalmol. 118 1994 445 450
19. A. Kvanta Subfoveal fibrovascular membranes in age-related macular degeneration express vascular endothelial growth factor Investigative Ophthalmology & Visual Science 37 9 1996 1929 1934
20. A.K. Olsson VEGF receptor signalling - in control of vascular function Nat. Rev. Mol. Cell Biol. 7 2006 359 371
21. N. Ferrara Vascular endothelial growth factor: basic science and clinical progress Endocr. Rev. 25 2004 581 611
22. W.Y. Shen Preclinical evaluation of a phosphorothioate oligonucleotide in the retina of rhesus monkey Lab. Invest. 82 2002 167 182
23. H. Kourlas, and D.S. Schiller Pegaptanib sodium for the treatment of neovascular age-related macular degeneration: a review Clin. Ther. 28 2006 36 44
24. M. Murata Inhibition of ocular angiogenesis by diced small interfering RNAs (siRNAs) specific to vascular endothelial growth factor (VEGF) Curr. Eye Res. 31 2006 171 180
25. C. Hecquet Activation and role of MAP kinase-dependent pathways in retinal pigment epithelial cells: ERK and RPE cell proliferation Invest. Ophthalmol. Vis. Sci. 43 2002 3091 3098
26. C.-H. Heldin, and B. Westermark Mechanism of Action and In Vivo Role of Platelet-Derived Growth Factor Physiological Reviews 79 4 1999 1283 1316
27. N. Jo Inhibition of platelet-derived growth factor B signaling enhances the efficacy of anti-vascular endothelial growth factor therapy in multiple models of ocular neovascularization Am. J. Pathol. 168 2006 2036 2053
28. M.F. Cordeiro Molecular therapy in ocular wound healing Br. J. Ophthalmol. 83 1999 1219 1224
29. M.F. Cordeiro Role of transforming growth factor beta in conjunctival scarring Clin. Sci. 104 2003 181 187
30. M.F. Cordeiro Novel antisense oligonucleotides targeting TGF-beta inhibit in vivo scarring and improve surgical outcome Gene Ther. 10 2003 59 71
31. H. Nakamura RNA interference targeting transforming growth factor-beta type II receptor suppresses ocular inflammation and fibrosis Mol. Vis. 10 2004 703 711
32. T.G. McCauley Pharmacologic and pharmacokinetic assessment of anti-TGFbeta2 aptamers in rabbit plasma and aqueous humor Pharm. Res. 23 2006 303 311
33. L. Desire Endogenous and exogenous fibroblast growth factor 2 support survival of chick retinal neurons by control of neuronal bcl-x(L) and bcl-2 expression through a fibroblast growth factor receptor 1- and ERK-dependent pathway J. Neurochem. 75 2000 151 163
34. S. Roy Reduction of fibronectin expression by intravitreal administration of antisense oligonucleotides Nat. Biotechnol. 17 1999 476 479
35. C. Lofqvist Quantification and localization of the IGF/insulin system expression in retinal blood vessels and neurons during oxygen-induced retinopathy in mice Invest. Ophthal. Vis. Sci. 50 2009 1831 1837
36. R.F. Gariano, and T.W. Gardner Retinal angiogenesis in development and disease Nature 438 2005 960 966
37. T.S. Kern Contributions of inflammatory processes to the development of the early stages of diabetic retinopathy Exp. Diabetes Res. 2007 2007 95103
38. Q. Zhang Suppression of retinal neovascularization by the iNOS inhibitor aminoguanidine in mice of oxygen-induced retinopathy Graefes. Arch. Clin. Exp. Ophthalmol. 247 2009 919 927
39. R. Naruse Oxidative stress and antioxidative potency are closely associated with diabetic retinopathy and nephropathy in patients with type 2 diabetes Saudi Med. J. 34 2013 135 141
40. Y. Nishizuka The role of protein kinase C in cell surface signal transduction and tumour promotion Nature 308 1984 693 698
41. L.P. Aiello The potential role of PKC β in diabetic retinopathy and macular edema Survey Ophthalmol. 47 Suppl. 2 2002 263 269
42. M.D. Davis Effect of ruboxistaurin on the visual acuity decline associated with long-standing diabetic macular edema Invest. Ophthalmol. Vis. Sci. 50 2009 1 4
43. D. Fabbro PKC412-a protein kinase inhibitor with a broad therapeutic potential Anti-Cancer Drug Des. 15 2000 17 28
44. O. Konopatskaya, and A.W. Poole Protein kinase Cα: disease regulator and therapeutic target Trends Pharmacol. Sci. 31 2010 8 14
45. S. Sivaprasad, and N.V. Chong The complement system and age-related macular degeneration Eye 20 2006 867 872
46. S. Khandhadia Age-related macular degeneration and the complement system Immunobiology 217 2012 127 146
47. D. Ricklin, and J.D. Lambris Compstatin: a complement inhibitor on its way to clinical application Adv. Exp. Med. Biol. 632 2008 273 292
48. J. Schubert Eculizumab, a terminal complement inhibitor, improves anaemia in patients with paroxysmal nocturnal haemoglobinuria Br. J. Haematol. 142 2008 263 272
49. L.S. Green Inhibitory DNA ligands to platelet-derived growth factor B-chain Biochemistry 35 1996 14413 14424
50. J.C. Gilbert First-in-human evaluation of anti-von Willebrand factor therapeutic aptamer ARC1779 in healthy volunteers Circulation 116 2007 2678 2686
51. L.L. Lebruska, and L.J. Maher Selection and characterization of an RNA decoy for transcription factor NF-κB Biochemistry 38 1999 3168 3174
52. B.J. Hicke Tenascin-C aptamers are generated using tumor cells and purified protein J. Biol. Chem. 276 2001 48644 48654
53. L.S. Green Nuclease-resistant nucleic acid ligands to vascular permeability factor/vascular endothelial growth factor Chem. Biol. 2 1995 683 695
54. S.E. Lupold Identification and characterization of nuclease-stabilized RNA molecules that bind human prostate cancer cells via the prostate-specific membrane antigen Cancer Res. 62 2002 4029 4033
55. A.C. Bird An international classification and grading system for age-related maculopathy and age-related macular degeneration Survey Ophthalmol. 39 1995 367 374
56. A. Amini The critical role of vascular endothelial growth factor in tumor angiogenesis Curr. Cancer Drug Targets 12 2012 23 43
57. N. Ferrara Vascular endothelial growth factor: basic science and clinical progress Endocrine Rev. 25 2004 581 611
58. T. Alon Vascular endothelial growth-factor acts as a survival factor for newly formed retinal-vessels and has implications for retinopathy of prematurity Nat. Med. 1 1995 1024 1028
59. J. Ruckman 2′-Fluoropyrimidine RNA-based aptamers to the 165-amino acid form of vascular endothelial growth factor (VEGF165): inhibition of receptor binding and VEGF-induced vascular permeability through interactions requiring the exon 7-encoded domain J. Biol. Chem. 273 1998 20556 20567
60. D. Jellinek Inhibition of receptor binding by high-affinity RNA ligands to vascular endothelial growth factor Biochemistry 33 1994 10450 10456
61. Eyetech Study Group Preclinical and Phase 1A clinical evaluation of an anti-VEGF pegylated aptamer (EYE001) for the treatment of exudative age-related macular degeneration Retina 22 2002 143 152
62. C.E. Tucker Detection and plasma pharmacokinetics of an anti-vascular endothelial growth factor oligonucleotide-aptamer (NX1838) in rhesus monkeys J. Chromatogr. B: Biomed. Sci. Appl. 732 1999 203 212
63. D. Drolet Pharmacokinetics and safety of an anti-vascular endothelial growth factor aptamer (NX1838) following injection into the vitreous humor of Rhesus monkeys Pharma. Res. 17 2000 1503 1510
64. C. Zehetner Plasma levels of vascular endothelial growth factor before and after intravitreal injection of bevacizumab, ranibizumab and pegaptanib in patients with age-related macular degeneration, and in patients with diabetic macular oedema Br. J. Ophthalmol. 97 2013 454 459
65. E.S. Gragoudas Pegaptanib for neovascular age-related macular degeneration N. Eng. J. Med. 351 2004 2805 2816
66. E.W. Ng, and A.P. Adamis Targeting angiogenesis, the underlying disorder in neovascular age-related macular degeneration Can. J. Ophthalmol. 40 2005 352 368
67. C.R. Gonzales Enhanced efficacy associated with early treatment of neovascular age-related macular degeneration with pegaptanib sodium: an exploratory analysis Retina 25 2005 815 827
68. E.T. Cunningham A Phase II randomized double-masked trial of pegaptanib, an anti-vascular endothelial growth factor aptamer, for diabetic macular edema Ophthalmology 112 2005 1747 1757
69. G. Querques Intravitreal pegaptanib sodium (Macugen) for diabetic macular oedema Acta Ophthalmol. 87 2009 623 630
70. M. Rinaldi Intravitreal pegaptanib sodium (Macugen®) for treatment of diabetic macular oedema: a morphologic and functional study Br. J. Clin. Pharmacol. 74 2012 940 946
71. G. Querques Intravitreal pegaptanib sodium (Macugen) for refractory cystoid macular edema in pericentral retinitis pigmentosa Int. Ophthalmol. 29 2009 103 107
72. C. Shima Complications in patients after intravitreal injection of bevacizumab Acta Ophthalmol. 86 2008 372 376
73. D. D'Amico, and A. Bird VEGF inhibition study in ocular neovascularization-1 (VISION-1): safety evaluation from the pivotal Macugen™ (pegaptanib sodium) clinical trials Invest. Ophtalmol. Vis. Sci. 45 2004 2363
74. M.S. Mulligan C5a-dependent up-regulation in vivo of lung vascular P-selectin J. Immunol. 158 1997 1857 1861
75. G. Biesecker Derivation of RNA aptamer inhibitors of human complement C5 Immunopharmacology 42 1999 219 230
76. Epstein, D. and Kurz, J.C. (2007) Complement Binding Aptamers and Anti-c5 Agents Useful in the Treatment of Ocular Disorders. Google Patents
77. O.S.G. Cousins Targeting complement factor 5 in combination with vascular endothelial growth factor (VEGF) inhibition for neovascular age related macular degeneration (AMD): results of a phase 1 study Arvo Meeting Abstracts 51 2010 1251
78. Benedict, C. et al. (2010) Aptamer Therapeutics Useful in the Treatment of Complement-related Disorders. Google Patents
79. J. Bradley Combination therapy for the treatment of ocular neovascularization Angiogenesis 10 2007 141 148
80. Shima, D. et al. (2012) Combination Therapy for the Treatment of Ocular Neovascular Disorders. EP Patent 1,660,057
81. M.A. Zarbin, and P.J. Rosenfeld Pathway-based therapies for age-related macular degeneration: an integrated survey of emerging treatment alternatives Retina 30 2010 1350 1367
82. P. Sundaram Therapeutic RNA aptamers in clinical trials Eur. J. Pharm. Sci. 48 2013 259 271
83. A. Tandon Role of transforming growth factor Beta in corneal function, biology and pathology Curr. Mol. Med. 10 2010 565 578
84. Toothman, P.J. et al. (1998) High Affinity TGF beta Nucleic Acid Ligands and Inhibitors. Google Patents
85. Pagratis, N. et al. (2002) High Affinity TGF. Google Patents
86. T. McCauley Pharmacologic and pharmacokinetic assessment of anti-TGFβ2 aptamers in rabbit plasma and aqueous humor Pharm. Res. 23 2006 303 311
87. D. Jellinek High-affinity RNA ligands to basic fibroblast growth factor inhibit receptor binding Proc. Natl. Acad. Sci. U. S. A. 90 1993 11227 11231
88. R.D. Jenison Oligonucleotide inhibitors of P-selectin-dependent neutrophil-platelet adhesion Antisense Nucleic Acid Drug Dev. 8 1998 265 279
89. J. Mi Targeted inhibition of alphavbeta3 integrin with an RNA aptamer impairs endothelial cell growth and survival Biochem. Biophys. Res. Commun. 338 2005 956 963
90. R.R. White A nuclease-resistant RNA aptamer specifically inhibits angiopoietin-1-mediated Tie2 activation and function Angiogenesis 11 2008 395 401
91. R.R. White Inhibition of rat corneal angiogenesis by a nuclease-resistant RNA aptamer specific for angiopoietin-2 Proc. Natl. Acad. Sci. U. S. A. 100 2003 5028 5033
92. X. Zhu A novel aptamer targeting TGF-β receptor II inhibits transdifferentiation of human Tenon's fibroblasts into myofibroblast Invest. Ophthalmol. Vis. Sci. 53 2012 6897 6903
93. Y. Nonaka Screening and improvement of an anti-VEGF DNA aptamer Molecules 15 2010 215 225
94. Y. Nonaka Electrochemical detection of vascular endothelial growth factor with aptamer sandwich Electrochemistry 80 2012 363 366
95. D.W. Drolet Pharmacokinetics and safety of an anti-vascular endothelial growth factor aptamer (NX1838) following injection into the vitreous humor of rhesus monkeys Pharm. Res. 17 2000 1503 1510
96. L. Griffin In vivo anticoagulant properties of a novel nucleotide-based thrombin inhibitor and demonstration of regional anticoagulation in extracorporeal circuits Blood 81 1993 3271 3276
97. E. Uhlmann Use of minimally modified antisense oligonucleotides for specific inhibition of gene expression Methods Enzymol. 313 2000 268 284
98. P.E. Burmeister Direct in vitro selection of a 2′-O-methyl aptamer to VEGF Chem. Biol. 12 2005 25 33
99. S.K. Singh LNA (locked nucleic acids): synthesis and high-affinity nucleic acid recognition Chem. Commun. 1998 1998 455 456
100. L. Crouzier Efficient reverse transcription using locked nucleic acid nucleotides towards the evolution of nuclease resistant RNA aptamers PLoS One 7 2012 e35990
101. C. Hull Effects of locked nucleic acid substitutions on the stability of oligonucleotide hairpins Nucleosides Nucleotides Nucleic Acids 31 2012 28 41
102. L.H. Lauridsen Enzymatic recognition of 2′-modified ribonucleoside 5′-triphosphates: towards the evolution of versatile aptamers ChemBioChem 13 2012 19 25
103. A.D. Keefe Aptamers as therapeutics Nat. Rev. Drug Discov. 9 2010 537 550
104. S. Mishra PEGylation significantly affects cellular uptake and intracellular trafficking of non-viral gene delivery particles Eur. J. Cell Biol. 83 2004 97 111
105. C.A. Farman, and D.J. Kornbrust Oligodeoxynucleotide studies in primates: antisense and immune stimulatory indications Toxicologic. Pathol. 31 Suppl. 1 2003 119 122
106. J.K. Armstrong Antibody against poly (ethylene glycol) adversely affects PEG-asparaginase therapy in acute lymphoblastic leukemia patients Cancer 110 2007 103 111
107. C.L. Cooper Immunostimulatory effects of three classes of CpG oligodeoxynucleotides on PBMC from HCV chronic carriers J. Immune Based Ther. Vaccines 6 2008 3
108. A.M. Krieg Toll-like receptor 9 (TLR9) agonists in the treatment of cancer Oncogene 27 2008 161 167
109. J.R. Kanwar Chimeric aptamers in cancer cell-targeted drug delivery Crit. Rev. Biochem. Mol. Biol. 46 2011 459 477
110. N. Subramanian Target-specific delivery of doxorubicin to retinoblastoma using epithelial cell adhesion molecule aptamer Mol. Vis. 18 2012 2783 2795
111. R. Gaudana Ocular drug delivery AAPS J. 12 2010 348 360
112. S.K. Sahoo Nanotechnology in ocular drug delivery Drug Discov. Today 13 2008 144 151
113. J.R. Kanwar Applications of aptamers in nanodelivery systems in cancer, eye and inflammatory diseases Nanomedicine 5 2010 1435 1445
114. R.N. Veedu, and J. Wengel Locked nucleic acid nucleoside triphosphates and polymerases: on the way towards evolution of LNA aptamers Mol. Biosyst. 5 2009 787 792
115. A.D. Burnette RNA aptamer therapy for vaso-occlusion in sickle cell disease Nucleic Acid Ther. 21 2011 275 283
116. B. Sennino Sequential loss of tumor vessel pericytes and endothelial cells after inhibition of platelet-derived growth factor B by selective aptamer AX102 Cancer Res. 67 2007 7358 7367
117. A.D. Keefe, and S.T. Cload SELEX with modified nucleotides Curr. Opin. Chem. Biol. 12 2008 448 456
118. J. Kang Combinatorial selection of a single stranded DNA thioaptamer targeting TGF-beta1 protein Bioorg. Med. Chem. Lett. 18 2008 1835 1839
119. A. Berezhnoy Thermal stability of siRNA modulates aptamer-conjugated siRNA inhibition Mol. Ther. Nucleic Acids 1 2012 e51
120. D.M. Dupont Serum-stable RNA aptamers to urokinase-type plasminogen activator blocking receptor binding RNA 16 2010 2360 2369
121. K. Fauster 2′-Azido RNA, a versatile tool for chemical biology: synthesis, X-ray structure, siRNA applications, click labeling ACS Chem. Biol. 7 2012 581 589
122. A. Nolte Mirror-design of L-oligonucleotide ligands binding to L-arginine Nat. Biotechnol. 14 1996 1116 1119
123. A. Vater A mixed mirror-image DNA/RNA aptamer inhibits glucagon and acutely improves glucose tolerance in models of type 1 and type 2 diabetes J. Biol. Chem. 288 2013 21136 21147
124. J. Soutschek Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs Nature 432 2004 173 178
125. K.K. Karlsen, and J. Wengel Locked nucleic acid and aptamers Nucleic Acid Ther. 22 2012 366 370
126. J.R. Kanwar Cancer targeted nanoparticles specifically induce apoptosis in cancer cells and spare normal cells Aus. J. Chem. 65 2012 5 14.