Therapeutic Oligonucleotides with Polyethylene Glycol Modifications

References

• Yamamoto T , NakataniM, NarukawaK, ObikaS. Antisense drug discovery and development. Future Med. Chem.3 (3), 339–365 (2011).
   PubMed Web of Science ®Google Scholar
• Dirin M , WinklerJ. Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides. Expert Opin. Biol. Ther.13 (6), 875–888 (2013).
   PubMed Web of Science ®Google Scholar
• Winkler J , StesslM, AmarteyJ, NoeCR. Off-target effects related to the phosphorothioate modification of nucleic acids. ChemMedChem5 (8), 1344–1352 (2010).
   PubMed Web of Science ®Google Scholar
• Geary RS . Antisense oligonucleotide pharmacokinetics and metabolism. Expert Opin. Drug Metab. Toxicol.5 (4), 381–391 (2009).
   PubMed Web of Science ®Google Scholar
• Cirak S , Arechavala-GomezaV, GuglieriMet al. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, Phase 2, dose-escalation study. Lancet378 (9791), 595–605 (2011).
   PubMed Web of Science ®Google Scholar
• Goemans NM , TuliniusM, van den AkkerJTet al. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N. Engl. J. Med.364 (16), 1513–1522 (2011).
   PubMed Web of Science ®Google Scholar
• Voit T , TopalogluH, StraubVet al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled Phase 2 study. Lancet Neurol.13 (10), 987–996 (2014).
   PubMed Web of Science ®Google Scholar
• Burnett JC , RossiJJ. RNA-based therapeutics: current progress and future prospects. Chem. Biol.19 (1), 60–71 (2012).
   PubMed Web of Science ®Google Scholar
• Lorenzer C , DirinM, WinklerA-M, BaumannV, WinklerJ. Going beyond the liver: progress and challenges of targeted delivery of siRNA therapeutics. J. Control. Release203 (1), 1–15 (2015).
   PubMedGoogle Scholar
• Chen S , BlankJL, PetersTet al. Genome-wide siRNA screen for modulators of cell death induced by proteasome inhibitor bortezomib. Cancer Res.70 (11), 4318–4326 (2010).
   PubMed Web of Science ®Google Scholar
• Whitehead KA , LangerR, AndersonDG. Knocking down barriers: advances in siRNA delivery. Nat. Rev. Drug Discov.8 (2), 129–138 (2009).
   PubMed Web of Science ®Google Scholar
• Manjunath N , DykxhoornDM. Advances in synthetic siRNA delivery. Discov. Med.9 (48), 418–430 (2010).
   PubMed Web of Science ®Google Scholar
• Semple SC , AkincA, ChenJet al. Rational design of cationic lipids for siRNA delivery. Nat. Biotechnol.28 (2), 172–176 (2010).
   PubMed Web of Science ®Google Scholar
• Aliabadi HM , LandryB, SunC, TangT, UludağH. Supramolecular assemblies in functional siRNA delivery: where do we stand?Biomaterials33 (8), 2546–2569 (2012).
   PubMed Web of Science ®Google Scholar
• Winkler J . Oligonucleotide conjugates for therapeutic applications. Ther. Deliv.4 (7), 791–809 (2013).
   PubMedGoogle Scholar
• Gomes-da-Silva LC , FonsecaNA, MouraV, Pedroso de LimaMC, SimoesS, MoreiraJN. Lipid-based nanoparticles for siRNA delivery in cancer therapy: paradigms and challenges. Acc. Chem. Res.45 (7), 1163–1171 (2012).
   PubMed Web of Science ®Google Scholar
• Akinc A , QuerbesW, DeSet al. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol. Ther.18 (7), 1357–1364 (2010).
   PubMed Web of Science ®Google Scholar
• Keefe AD , PaiS, EllingtonA. Aptamers as therapeutics. Nat. Rev. Drug Discov.9 (7), 537–550 (2010).
   PubMed Web of Science ®Google Scholar
• Ng EWM , ShimaDT, CaliasP, CunninghamETJr, GuyerDR, AdamisAP. Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease. Nat. Rev. Drug Discov.5 (2), 123–132 (2006).
   PubMed Web of Science ®Google Scholar
• Lindow M , KauppinenS. Discovering the first microRNA-targeted drug. J. Cell Biol.199 (3), 407–412 (2012).
   PubMed Web of Science ®Google Scholar
• Baumann V , WinklerJ. miRNA-based therapies: strategies and delivery platforms for oligonucleotide and non-oligonucleotide agents. Future Med. Chem.6 (17), 1967–1984 (2014).
   PubMed Web of Science ®Google Scholar
• van Rooij E , OlsonEN. MicroRNA therapeutics for cardiovascular disease: opportunities and obstacles. Nat. Rev. Drug Discov.11 (11), 860–872 (2012).
   PubMed Web of Science ®Google Scholar
• Sassen S , MiskaEA, CaldasC. MicroRNA: implications for cancer. Virchows Arch.452 (1), 1–10 (2008).
   PubMed Web of Science ®Google Scholar
• Yeung ML , JeangKT. MicroRNAs and cancer therapeutics. Pharm. Res.28 (12), 3043–3049 (2011).
   PubMed Web of Science ®Google Scholar
• Pritchard CC , KrohE, WoodBet al. Blood cell origin of circulating microRNAs: a cautionary note for cancer biomarker studies. Cancer Prev. Res.5 (3), 492–497 (2012).
   PubMed Web of Science ®Google Scholar
• Ibrahim Ahmed G-E , ChengK, MarbánE. Exosomes as critical agents of cardiac regeneration triggered by cell therapy. Stem Cell Reports2 (5), 606–619 (2014).
   PubMed Web of Science ®Google Scholar
• Bagnall RD , TsoutsmanT, ShephardRE, RitchieW, SemsarianC. Global microRNA profiling of the mouse ventricles during development of severe hypertrophic cardiomyopathy and heart failure. PLoS ONE7 (9), e44744 (2012).
   PubMed Web of Science ®Google Scholar
• Janssen HLA , ReesinkHW, LawitzEJet al. Treatment of HCV infection by targeting microRNA. N. Engl. J. Med.368 (18), 1685–1694 (2013).
   PubMed Web of Science ®Google Scholar
• Gebert LFR , RebhanMAE, CrivelliSEM, DenzlerR, StoffelM, HallJ. Miravirsen (SPC3649) can inhibit the biogenesis of miR-122. Nucleic Acids Res.42 (1), 609–621 (2014).
   PubMed Web of Science ®Google Scholar
• Liu C , KelnarK, LiuBet al. The microRNA miR-34a inhibits prostate cancer stem cells and metastasis by directly repressing CD44. Nat. Med.17 (2), 211–215 (2011).
   PubMed Web of Science ®Google Scholar
• Veronese FM , MeroA. The Impact of PEGylation on biological therapies. BioDrugs22 (5), 315–329 (2008).
   PubMed Web of Science ®Google Scholar
• Hadziyannis SJ , PapatheodoridisGV. Peginterferon-α2a (40 kDa) for chronic hepatitis C. Expert Opin. Pharmacother.4 (4), 541–551 (2003).
   PubMed Web of Science ®Google Scholar
• Bukowski RM , TendlerC, CutlerD, RoseE, LaughlinMM, StatkevichP. Treating cancer with PEG intron. Cancer95 (2), 389–396 (2002).
   PubMedGoogle Scholar
• Topf JM . CERA: third-generation erythropoiesis-stimulating agent. Expert Opin. Pharmacother.9 (5), 839–849 (2008).
   PubMed Web of Science ®Google Scholar
• Milla P , DosioF, CattelL. PEGylation of proteins and liposomes: a powerful and flexible strategy to improve the drug delivery. Curr. Drug Metab.13 (1), 105–119 (2012).
   PubMed Web of Science ®Google Scholar
• Palm T , EsfandiaryR, GandhiR. The effect of PEGylation on the stability of small therapeutic proteins. Pharm. Dev. Technol.16 (5), 441–448 (2011).
   PubMed Web of Science ®Google Scholar
• Jevsevar S , KunsteljM, PorekarVG. PEGylation of therapeutic proteins. Biotechnol. J.5 (1), 113–128 (2010).
   PubMed Web of Science ®Google Scholar
• Mu Q , HuT, YuJ. Molecular insight into the steric shielding effect of PEG on the conjugated staphylokinase: biochemical characterization and molecular dynamics simulation. PLOS ONE8 (7), e68559 (2013).
   PubMed Web of Science ®Google Scholar
• Immordino ML , DosioF, CattelL. Stealth liposomes: review of the basic science, rationale, and clinical applications, existing and potential. Int. J. Nanomed.1 (3), 297–315 (2006).
   PubMed Web of Science ®Google Scholar
• Lorusso D , Di StefanoA, CaroneV, FagottiA, PiscontiS, ScambiaG. Pegylated liposomal doxorubicin-related palmar-plantar erythrodysesthesia (‘hand–foot’ syndrome). Ann. Oncol.18 (7), 1159–1164 (2007).
   PubMed Web of Science ®Google Scholar
• Sherman MR , WilliamsLD, SobczykMA, MichaelsSJ, SaiferMGP. Role of the methoxy group in immune responses to mPEG-protein conjugates. Bioconjug. Chem.23 (3), 485–499 (2012).
   PubMed Web of Science ®Google Scholar
• Armstrong JK , HempelG, KolingSet al. Antibody against poly(ethylene glycol) adversely affects PEG-asparaginase therapy in acute lymphoblastic leukemia patients. Cancer110 (1), 103–111 (2007).
   PubMed Web of Science ®Google Scholar
• Matsuda F , ToriiY, EnomotoHet al. Anti-interferon-α neutralizing antibody is associated with nonresponse to pegylated interferon-α plus ribavirin in chronic hepatitis C. J. Viral Hepat.19 (10), 694–703 (2012).
   PubMed Web of Science ®Google Scholar
• Saifer MGP , WilliamsLD, SobczykMA, MichaelsSJ, ShermanMR. Selectivity of binding of PEGs and PEG-like oligomers to anti-PEG antibodies induced by methoxyPEG-proteins. Mol. Immunol.57 (2), 236–246 (2014).
   PubMed Web of Science ®Google Scholar
• Jäschke A , FürsteJP, NordhoffE, HillenkampF, CechD, ErdmannVA. Synthesis and properties of oligodeoxyribonucleotide – polyethylene glycol conjugates. Nucleic Acids Res.22 (22), 4810–4817 (1994).
   PubMed Web of Science ®Google Scholar
• Lallana E , Sousa-HervesA, Fernandez-TrilloF, RigueraR, Fernandez-MegiaE. Click chemistry for drug delivery nanosystems. Pharm. Res.29 (1), 1–34 (2012).
   PubMed Web of Science ®Google Scholar
• Ming X , LaingB. Bioconjugates for targeted delivery of therapeutic oligonucleotides. Adv. Drug Deliv. Rev. (0)).
   Google Scholar
• Ikeda Y , NagasakiY. Impacts of PEGylation on the gene and oligonucleotide delivery system. J. App. Polymer Sci.131 (9), n/a–n/a (2014).
   Web of Science ®Google Scholar
• Graham MJ , CrookeST, MonteithDKet al. In vivo distribution and metabolism of a phosphorothioate oligonucleotide within rat liver after intravenous administration. J. Pharmacol. Exp. Ther.286 (1), 447–458 (1998).
   PubMed Web of Science ®Google Scholar
• Lonnberg H . Solid-phase synthesis of oligonucleotide conjugates useful for delivery and targeting of potential nucleic acid therapeutics. Bioconjug. Chem.20 (6), 1065–1094 (2009).
   PubMed Web of Science ®Google Scholar
• Jung S , LeeSH, MokH, ChungHJ, ParkTG. Gene silencing efficiency of siRNA-PEG conjugates: effect of PEGylation site and PEG molecular weight. J. Control. Release144 (3), 306–313 (2010).
   PubMed Web of Science ®Google Scholar
• Iversen F , YangC, Dagnæs-HansenF, SchaffertDH, KjemsJ, GaoS. Optimized siRNA-PEG conjugates for extended blood circulation and reduced urine excretion in mice. Theranostics3 (3), 201–209 (2013).
   PubMed Web of Science ®Google Scholar
• Gaziova Z , BaumannV, WinklerA-M, WinklerJ. Chemically defined polyethylene glycol siRNA conjugates with enhanced gene silencing effect. Bioorg. Med. Chem.22 (7), 2320–2326 (2014).
   PubMed Web of Science ®Google Scholar
• Bramsen JB , LaursenMB, NielsenAFet al. A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity. Nucleic Acids Res.37 (9), 2867–2881 (2009).
   PubMed Web of Science ®Google Scholar
• Eckstein F . Phosphorothioates, essential components of therapeutic oligonucleotides. Nucl. Acids Therap.24 (6), 374–387 (2014).
   PubMed Web of Science ®Google Scholar
• Winkler J , GilbertM, KocourkováA, StesslM, NoeCR. 2′-O-Lysylaminohexyl oligonucleotides: modifications for antisense and siRNA. ChemMedChem3 (1), 102–110 (2008).
   PubMed Web of Science ®Google Scholar
• Werner D , BrunarH, NoeCR. Investigations on the influence of 2′-O-alkyl modifications on the base pairing properties of oligonucleotides. Pharm. Acta Helv.73 (1), 3–10 (1998).
   Google Scholar
• Patinkin D , HidmiA, WeissL, SlavinS, KatzhendlerJ. The effect of pegylated antisense acetylcholinesterase on hematopoiesis. Oligonucleotides13 (4), 207–216 (2003).
   PubMedGoogle Scholar
• Rapozzi V , CogoiS, SpessottoPet al. Antigene effect in K562 cells of a PEG-conjugated triplex-forming oligonucleotide targeted to the bcr/abl oncogene. Biochemistry41 (2), 502–510 (2002).
   PubMed Web of Science ®Google Scholar
• Zhao H , PengP, LongleyCet al. Delivery of G3139 using releasable PEG-linkers: impact on pharmacokinetic profile and anti-tumor efficacy. J. Control. Release119 (2), 143–152 (2007).
   PubMed Web of Science ®Google Scholar
• Shokrzadeh N , WinklerA-M, DirinM, WinklerJ. Oligonucleotides conjugated with short chemically defined polyethylene glycol chains are efficient antisense agents. Bioorg. Med. Chem. Lett.24 (24), 5758–5761 (2014).
   PubMed Web of Science ®Google Scholar
• Vinores SA . Pegaptanib in the treatment of wet, age-related macular degeneration. Int. J. Nanomed.1 (3), 263–268 (2006).
   PubMed Web of Science ®Google Scholar
• Rosina C , BottoniF, StaurenghiG. Clinical experience with pegaptanib sodium. Clin. Ophthalmol.2 (3), 485–488 (2008).
   PubMedGoogle Scholar
• Ni X , CastanaresM, MukherjeeA, LupoldSE. Nucleic acid aptamers: clinical applications and promising new horizons. Curr. Med. Chem.18 (27), 4206–4214 (2011).
   PubMed Web of Science ®Google Scholar
• Vavalle JP , CohenMG. The REG1 anticoagulation system: a novel actively controlled factor IX inhibitor using RNA aptamer technology for treatment of acute coronary syndrome. Future Cardiol.8 (3), 371–382 (2012).
   PubMedGoogle Scholar
• Povsic TJ , VavalleJP, AberleLHet al. A Phase 2, randomized, partially blinded, active-controlled study assessing the efficacy and safety of variable anticoagulation reversal using the REG1 system in patients with acute coronary syndromes: results of the RADAR trial. Eur. Heart J.34 (31), 2481–2489 (2013).
   PubMed Web of Science ®Google Scholar
• Hoellenriegel J , ZboralskiD, MaaschCet al. The Spiegelmer NOX-A12, a novel CXCL12 inhibitor, interferes with chronic lymphocytic leukemia cell motility and causes chemosensitization. Blood123 (7), 1032–1039 (2014).
   PubMed Web of Science ®Google Scholar
• Vater A , KlussmannS. Turning mirror-image oligonucleotides into drugs: the evolution of Spiegelmer® therapeutics. Drug Discov. Today20 (1), 147–155 (2015).
   PubMed Web of Science ®Google Scholar
• Vater A , SahlmannJ, KrogerNet al. Hematopoietic stem and progenitor cell mobilization in mice and humans by a first-in-class mirror-image oligonucleotide inhibitor of CXCL12. Clin. Pharmacol. Ther.94 (1), 150–157 (2013).
   PubMed Web of Science ®Google Scholar
• Chow FY , Nikolic-PatersonDJ, OzolsE, AtkinsRC, RollinBJ, TeschGH. Monocyte chemoattractant protein-1 promotes the development of diabetic renal injury in streptozotocin-treated mice. Kidney Int.69 (1), 73–80 (2006).
   PubMed Web of Science ®Google Scholar
• van Eijk LT , JohnAS, SchwoebelFet al. Effect of the antihepcidin Spiegelmer lexaptepid on inflammation-induced decrease in serum iron in humans. Blood124 (17), 2643–2646 (2014).
   PubMed Web of Science ®Google Scholar
• Mayr FB , KnöblP, JilmaBet al. The aptamer ARC1779 blocks von Willebrand factor-dependent platelet function in patients with thrombotic thrombocytopenic purpura ex vivo. Transfusion (Paris).50 (5), 1079–1087 (2010).
   Web of Science ®Google Scholar
• Healy JM , LewisSD, KurzMet al. Pharmacokinetics and biodistribution of novel aptamer compositions. Pharm. Res.21 (12), 2234–2246 (2004).
   PubMed Web of Science ®Google Scholar
• Ricotta DN , FrishmanW. Mipomersen: a safe and effective antisense therapy adjunct to statins in patients with hypercholesterolemia. Cardiol. Rev.20 (2), 90–95 (2012).
   PubMed Web of Science ®Google Scholar
• Visser ME , WagenerG, BakerBFet al. Mipomersen, an apolipoprotein B synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: a randomized, double-blind, placebo-controlled trial. Eur. Heart J.33 (9), 1142–1149 (2012).
   PubMed Web of Science ®Google Scholar
• Saad F , HotteS, NorthSet al. Randomized Phase II trial of custirsen (OGX-011) in combination with docetaxel or mitoxantrone as second-line therapy in patients with metastatic castrate-resistant prostate cancer progressing after first-line docetaxel: CUOG Trial P-06c. Clin. Cancer Res.17 (17), 5765–5773 (2011).
   PubMed Web of Science ®Google Scholar
• Li S , FuJ, LentzschS. Cap-dependent protein translation initiation in multiple myeloma: an attractive target for therapy. In : Genetic and Molecular Epidemiology of Multiple Myeloma. LentzschS ( Ed.). Springer, NY, USA, 43–57 (2013).
   Google Scholar
• Noveck R , StroesESG, FlaimJDet al. Effects of an antisense oligonucleotide inhibitor of c‐reactive protein synthesis on the endotoxin challenge response in healthy human male volunteers. J. Am. Heart Assoc.3 (4), (2014).
   PubMed Web of Science ®Google Scholar
• Baylot V , AndrieuC, KatsogiannouMet al. OGX-427 inhibits tumor progression and enhances gemcitabine chemotherapy in pancreatic cancer. Cell Death Dis.2, e221 (2011).
   PubMed Web of Science ®Google Scholar
• Friberg TR , TolentinoM, WeberP, PatelS, CampbellS, GoldbaumM. Pegaptanib sodium as maintenance therapy in neovascular age-related macular degeneration: the LEVEL study. Br. J. Ophthalmol.94 (12), 1611–1617 (2010).
   PubMed Web of Science ®Google Scholar
• Gutsaeva DR , ParkersonJB, YerigenahallySDet al. Inhibition of cell adhesion by anti–P-selectin aptamer: a new potential therapeutic agent for sickle cell disease. Blood117 (2), 727–735 (2011).
   PubMed Web of Science ®Google Scholar
• Ishikawa M , JinD, SawadaY, AbeS, YoshitomiT. Future therapies of wet age-related macular degeneration. J. Ophthalmol.2015, 10 (2015).
   Web of Science ®Google Scholar