Nanoparticle for delivery of antisense γPNA oligomers targeting CCR5

References

• Paterson BM, Roberts BE, Kuff EL. Structural gene identification and mapping by DNA-mRNA hybrid-arrested cell-free translation. Proc Natl Acad Sci U S A 1977; 74:4370 - 4; http://dx.doi.org/10.1073/pnas.74.10.4370; PMID: 270678
   PubMed Web of Science ®Google Scholar
• Nielsen PE. Peptide nucleic acid. A molecule with two identities. Acc Chem Res 1999; 32:624 - 30; http://dx.doi.org/10.1021/ar980010t
   Web of Science ®Google Scholar
• Stein CA. Keeping the biotechnology of antisense in context. Nat Biotechnol 1999; 17:209; http://dx.doi.org/10.1038/6909; PMID: 10096271
   PubMed Web of Science ®Google Scholar
• Juliano RL, Yoo H. Aspects of the transport and delivery of antisense oligonucleotides. Curr Opin Mol Ther 2000; 2:297 - 303; PMID: 11249624
   PubMed Web of Science ®Google Scholar
• Deere J, Iversen P, Geller BL. Antisense phosphorodiamidate morpholino oligomer length and target position effects on gene-specific inhibition in Escherichia coli. Antimicrob Agents Chemother 2005; 49:249 - 55; http://dx.doi.org/10.1128/AAC.49.1.249-255.2005; PMID: 15616302
   PubMed Web of Science ®Google Scholar
• Summerton J, Stein D, Huang SB, Matthews P, Weller D, Partridge M. Morpholino and phosphorothioate antisense oligomers compared in cell-free and in-cell systems. Antisense Nucleic Acid Drug Dev 1997; 7:63 - 70; http://dx.doi.org/10.1089/oli.1.1997.7.63; PMID: 9149841
   PubMedGoogle Scholar
• Koshkin AA, Singh SK, Nielsen P, Rajwanshi VK, Kumar R, Meldgaard M, et al. LNA (Locked Nucleic Acids): Synthesis of the adenine, cytosine, guanine, 5-methylcytosine, thymine and uracil bicyclonucleoside monomers, oligomerisation, and unprecedented nucleic acid recognition. Tetrahedron 1998; 54:3607 - 30; http://dx.doi.org/10.1016/S0040-4020(98)00094-5
   Web of Science ®Google Scholar
• Nielsen PE, Egholm M, Berg RH, Buchardt O. Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide. Science 1991; 254:1497 - 500; http://dx.doi.org/10.1126/science.1962210; PMID: 1962210
   PubMed Web of Science ®Google Scholar
• Winssinger N, Ficarro S, Schultz PG, Harris JL. Profiling protein function with small molecule microarrays. Proc Natl Acad Sci U S A 2002; 99:11139 - 44; http://dx.doi.org/10.1073/pnas.172286899; PMID: 12167675
   PubMed Web of Science ®Google Scholar
• Debaene F, Da Silva JA, Pianowski Z, Duran FJ, Winssinger N. Expanding the scope of PNA-encoded libraries: divergent synthesis of libraries targeting cysteine, serine and metalloproteases as well as tyrosine phosphatases. Tetrahedron 2007; 63:6577 - 86; http://dx.doi.org/10.1016/j.tet.2007.03.033
   Web of Science ®Google Scholar
• Egholm M, Buchardt O, Nielsen PE, Berg RH. Peptide Nucleic-Acids (Pna) - Oligonucleotide Analogs with an Achiral Peptide Backbone. J Am Chem Soc 1992; 114:1895 - 7; http://dx.doi.org/10.1021/ja00031a062
   Web of Science ®Google Scholar
• Boyarskaya NP, Kirillova YG, Stotland EA, Prokhorov DI, Zvonkova EN, Shvets VI. Synthesis of two new thymine-containing negatively charged PNA monomers. Dokl Chem 2006; 408:57 - 60; http://dx.doi.org/10.1134/S0012500806050016
   Web of Science ®Google Scholar
• Hudson RHE, Liu YH, Wojciechowski F. Hydrophilic modifications in peptide nucleic acid - Synthesis and properties of PNA possessing 5-hydroxymethyluracil and 5-hydroxymethylcytosine. Can J Chem 2007; 85:302 - 12; http://dx.doi.org/10.1139/v07-030
   Web of Science ®Google Scholar
• Bonora GM, Drioli S, Ballico M, Faccini A, Corradini R, Cogoi S, et al. PNA conjugated to high-molecular weight poly(ethylene glycol): synthesis and properties. Nucleosides Nucleotides Nucleic Acids 2007; 26:661 - 4; http://dx.doi.org/10.1080/15257770701490548; PMID: 18066875
   PubMed Web of Science ®Google Scholar
• Dragulescu-Andrasi A, Rapireddy S, Frezza BM, Gayathri C, Gil RR, Ly DH. A simple gamma-backbone modification preorganizes peptide nucleic acid into a helical structure. J Am Chem Soc 2006; 128:10258 - 67; http://dx.doi.org/10.1021/ja0625576; PMID: 16881656
   PubMed Web of Science ®Google Scholar
• Rapireddy S, He G, Roy S, Armitage BA, Ly DH. Strand invasion of mixed-sequence B-DNA by acridine-linked, gamma-peptide nucleic acid (gamma-PNA). J Am Chem Soc 2007; 129:15596 - 600; http://dx.doi.org/10.1021/ja074886j; PMID: 18027941
   PubMed Web of Science ®Google Scholar
• Sahu B, Sacui I, Rapireddy S, Zanotti KJ, Bahal R, Armitage BA, et al. Synthesis and characterization of conformationally preorganized, (R)-diethylene glycol-containing γ-peptide nucleic acids with superior hybridization properties and water solubility. J Org Chem 2011; 76:5614 - 27; http://dx.doi.org/10.1021/jo200482d; PMID: 21619025
   PubMed Web of Science ®Google Scholar
• Rapireddy S, Bahal R, Ly DH. Strand invasion of mixed-sequence, double-helical B-DNA by γ-peptide nucleic acids containing G-clamp nucleobases under physiological conditions. Biochemistry 2011; 50:3913 - 8; http://dx.doi.org/10.1021/bi2002554; PMID: 21476606
   PubMed Web of Science ®Google Scholar
• He G, Rapireddy S, Bahal R, Sahu B, Ly DH. Strand invasion of extended, mixed-sequence B-DNA by gammaPNAs. J Am Chem Soc 2009; 131:12088 - 90; http://dx.doi.org/10.1021/ja900228j; PMID: 19663424
   PubMed Web of Science ®Google Scholar
• Crawford MJ, Rapireddy S, Bahal R, Sacui I, Ly DH. Effect of steric constraint at the γ-backbone position on the conformations and hybridization properties of PNAs. J Nucleic Acids 2011:652702, 10 pp.
   Google Scholar
• Bahal R, Sahu B, Rapireddy S, Lee CM, Ly DH. Sequence-unrestricted, Watson-Crick recognition of double helical B-DNA by (R)-miniPEG-γPNAs. Chembiochem 2012; 13:56 - 60; http://dx.doi.org/10.1002/cbic.201100646; PMID: 22135012
   PubMed Web of Science ®Google Scholar
• McNeer NA, Chin JY, Schleifman EB, Fields RJ, Glazer PM, Saltzman WM. Nanoparticles deliver triplex-forming PNAs for site-specific genomic recombination in CD34+ human hematopoietic progenitors. Mol Ther 2011; 19:172 - 80; http://dx.doi.org/10.1038/mt.2010.200; PMID: 20859257
   PubMed Web of Science ®Google Scholar
• McNeer NA, Schleifman EB, Cuthbert A, Brehm M, Jackson A, Cheng C, et al. Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo. Gene Ther 2012; 20:658 - 69; PMID: 23076379
   PubMed Web of Science ®Google Scholar
• Cheng CJ, Saltzman WM. Polymer nanoparticle-mediated delivery of microRNA inhibition and alternative splicing. Mol Pharm 2012; 9:1481 - 8; PMID: 22482958
   PubMed Web of Science ®Google Scholar
• Samson M, Libert F, Doranz BJ, Rucker J, Liesnard C, Farber CM, et al. Resistance to HIV-1 infection in caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene. Nature 1996; 382:722 - 5; http://dx.doi.org/10.1038/382722a0; PMID: 8751444
   PubMed Web of Science ®Google Scholar
• Schleifman EB, Bindra R, Leif J, del Campo J, Rogers FA, Uchil P, et al. Targeted disruption of the CCR5 gene in human hematopoietic stem cells stimulated by peptide nucleic acids. Chem Biol 2011; 18:1189 - 98; http://dx.doi.org/10.1016/j.chembiol.2011.07.010; PMID: 21944757
   PubMed Web of Science ®Google Scholar
• Christensen L, Fitzpatrick R, Gildea B, Petersen KH, Hansen HF, Koch T, et al. Solid-phase synthesis of peptide nucleic acids. J Pept Sci 1995; 1:175 - 83; http://dx.doi.org/10.1002/psc.310010304; PMID: 9222994
   PubMedGoogle Scholar
• Sahu B, Chenna V, Lathrop KL, Thomas SM, Zon G, Livak KJ, et al. Synthesis of conformationally preorganized and cell-permeable guanidine-based gamma-peptide nucleic acids (gammaGPNAs). J Org Chem 2009; 74:1509 - 16; http://dx.doi.org/10.1021/jo802211n; PMID: 19161276
   PubMed Web of Science ®Google Scholar
• Zhou P, Dragulescu-Andrasi A, Bhattacharya B, O’Keefe H, Vatta P, Hyldig-Nielsen JJ, et al. Synthesis of cell-permeable peptide nucleic acids and characterization of their hybridization and uptake properties. Bioorg Med Chem Lett 2006; 16:4931 - 5; http://dx.doi.org/10.1016/j.bmcl.2006.06.052; PMID: 16809033
   PubMed Web of Science ®Google Scholar
• Dragulescu-Andrasi A, Rapireddy S, He GF, Bhattacharya B, Hyldig-Nielsen JJ, Zon G, et al. Cell-permeable peptide nucleic acid designed to bind to the 5′-untranslated region of E-cadherin transcript induces potent and sequence-specific antisense effects. J Am Chem Soc 2006; 128:16104 - 12; http://dx.doi.org/10.1021/ja063383v; PMID: 17165763
   PubMed Web of Science ®Google Scholar
• Jagodzinski PP, Wierzbicki A, Wustner J, Kaneko Y, Kozbor D. Enhanced human immunodeficiency virus infection in macrophages by high-molecular-weight dextran sulfate is associated with conformational changes of gp120 and expression of the CCR5 receptor. Viral Immunol 1999; 12:23 - 33; http://dx.doi.org/10.1089/vim.1999.12.23; PMID: 10333239
   PubMed Web of Science ®Google Scholar
• Konopka K, Düzgüneş N. Expression of CD4 controls the susceptibility of THP-1 cells to infection by R5 and X4 HIV type 1 isolates. AIDS Res Hum Retroviruses 2002; 18:123 - 31; http://dx.doi.org/10.1089/08892220252779665; PMID: 11839145
   PubMed Web of Science ®Google Scholar
• Blum JS, Saltzman WM. High loading efficiency and tunable release of plasmid DNA encapsulated in submicron particles fabricated from PLGA conjugated with poly-L-lysine. J Control Release 2008; 129:66 - 72; http://dx.doi.org/10.1016/j.jconrel.2008.04.002; PMID: 18511145
   PubMed Web of Science ®Google Scholar
• Woodrow KA, Cu Y, Booth CJ, Saucier-Sawyer JK, Wood MJ, Saltzman WM. Intravaginal gene silencing using biodegradable polymer nanoparticles densely loaded with small-interfering RNA. Nat Mater 2009; 8:526 - 33; http://dx.doi.org/10.1038/nmat2444; PMID: 19404239
   PubMed Web of Science ®Google Scholar
• Babar IA, Cheng CJ, Booth CJ, Liang X, Weidhaas JB, Saltzman WM, et al. Nanoparticle-based therapy in an in vivo microRNA-155 (miR-155)-dependent mouse model of lymphoma. Author summary. Proc Natl Acad Sci USA 2012; 109:10140 - 1; http://dx.doi.org/10.1073/pnas.1201516109
   Web of Science ®Google Scholar
• Anderson J, Akkina R. Complete knockdown of CCR5 by lentiviral vector-expressed siRNAs and protection of transgenic macrophages against HIV-1 infection. Gene Ther 2007; 14:1287 - 97; http://dx.doi.org/10.1038/sj.gt.3302958; PMID: 17597795
   PubMed Web of Science ®Google Scholar
• Monia BP, Lesnik EA, Gonzalez C, Lima WF, McGee D, Guinosso CJ, et al. Evaluation of 2′-modified oligonucleotides containing 2′-deoxy gaps as antisense inhibitors of gene expression. J Biol Chem 1993; 268:14514 - 22; PMID: 8390996
   PubMed Web of Science ®Google Scholar
• Dias N, Stein CA. Antisense oligonucleotides: basic concepts and mechanisms. Mol Cancer Ther 2002; 1:347 - 55; PMID: 12489851
   PubMed Web of Science ®Google Scholar
• Tonkinson JL, Stein CA. Patterns of intracellular compartmentalization, trafficking and acidification of 5′-fluorescein labeled phosphodiester and phosphorothioate oligodeoxynucleotides in HL60 cells. Nucleic Acids Res 1994; 22:4268 - 75; http://dx.doi.org/10.1093/nar/22.20.4268; PMID: 7937155
   PubMed Web of Science ®Google Scholar
• Koziolkiewicz M, Gendaszewska E, Maszewska M, Stein CA, Stec WJ. The mononucleotide-dependent, nonantisense mechanism of action of phosphodiester and phosphorothioate oligonucleotides depends upon the activity of an ecto-5′-nucleotidase. Blood 2001; 98:995 - 1002; http://dx.doi.org/10.1182/blood.V98.4.995; PMID: 11493444
   PubMed Web of Science ®Google Scholar
• Guvakova MA, Yakubov LA, Vlodavsky I, Tonkinson JL, Stein CA. Phosphorothioate oligodeoxynucleotides bind to basic fibroblast growth factor, inhibit its binding to cell surface receptors, and remove it from low affinity binding sites on extracellular matrix. J Biol Chem 1995; 270:2620 - 7; http://dx.doi.org/10.1074/jbc.270.6.2620; PMID: 7852327
   PubMed Web of Science ®Google Scholar
• Larrouy B, Boiziau C, Sproat B, Toulmé J-J. RNase H is responsible for the non-specific inhibition of in vitro translation by 2′-O-alkyl chimeric oligonucleotides: high affinity or selectivity, a dilemma to design antisense oligomers. Nucleic Acids Res 1995; 23:3434 - 40; http://dx.doi.org/10.1093/nar/23.17.3434; PMID: 7567453
   PubMed Web of Science ®Google Scholar
• Hanvey JC, Peffer NJ, Bisi JE, Thomson SA, Cadilla R, Josey JA, et al. Antisense and antigene properties of peptide nucleic acids. Science 1992; 258:1481 - 5; http://dx.doi.org/10.1126/science.1279811; PMID: 1279811
   PubMed Web of Science ®Google Scholar
• Knudsen H, Nielsen PE. Antisense properties of duplex- and triplex-forming PNAs. Nucleic Acids Res 1996; 24:494 - 500; http://dx.doi.org/10.1093/nar/24.3.494; PMID: 8602363
   PubMed Web of Science ®Google Scholar
• Koppelhus U, Zachar V, Nielsen PE, Liu X, Eugen-Olsen J, Ebbesen P. Efficient in vitro inhibition of HIV-1 gag reverse transcription by peptide nucleic acid (PNA) at minimal ratios of PNA/RNA. Nucleic Acids Res 1997; 25:2167 - 73; http://dx.doi.org/10.1093/nar/25.11.2167; PMID: 9153317
   PubMed Web of Science ®Google Scholar
• Gambacorti-Passerini C, Mologni L, Bertazzoli C, le Coutre P, Marchesi E, Grignani F, et al. In vitro transcription and translation inhibition by anti-promyelocytic leukemia (PML)/retinoic acid receptor α and anti-PML peptide nucleic acid. Blood 1996; 88:1411 - 7; PMID: 8695861
   PubMed Web of Science ®Google Scholar
• Mologni L, Nielsen PE, Gambacorti-Passerini C. In vitro transcriptional and translational block of the bcl-2 gene operated by peptide nucleic acid. Biochem Biophys Res Commun 1999; 264:537 - 43; http://dx.doi.org/10.1006/bbrc.1999.1548; PMID: 10529398
   PubMed Web of Science ®Google Scholar
• Doyle DF, Braasch DA, Simmons CG, Janowski BA, Corey DR. Inhibition of gene expression inside cells by peptide nucleic acids: effect of mRNA target sequence, mismatched bases, and PNA length. Biochemistry 2001; 40:53 - 64; http://dx.doi.org/10.1021/bi0020630; PMID: 11141056
   PubMed Web of Science ®Google Scholar
• Chiarantini L, Cerasi A, Fraternale A, Andreoni F, Scarí S, Giovine M, et al. Inhibition of macrophage iNOS by selective targeting of antisense PNA. Biochemistry 2002; 41:8471 - 7; http://dx.doi.org/10.1021/bi020079f; PMID: 12081497
   PubMed Web of Science ®Google Scholar
• Boado RJ, Tsukamoto H, Pardridge WM. Drug delivery of antisense molecules to the brain for treatment of Alzheimer’s disease and cerebral AIDS. J Pharm Sci 1998; 87:1308 - 15; http://dx.doi.org/10.1021/js9800836; PMID: 9811482
   PubMed Web of Science ®Google Scholar
• Fraser GL, Holmgren J, Clarke PBS, Wahlestedt C. Antisense inhibition of δ-opioid receptor gene function in vivo by peptide nucleic acids. Mol Pharmacol 2000; 57:725 - 31; PMID: 10727518
   PubMed Web of Science ®Google Scholar
• Turner BJ, Cheah IK, Macfarlane KJ, Lopes EC, Petratos S, Langford SJ, et al. Antisense peptide nucleic acid-mediated knockdown of the p75 neurotrophin receptor delays motor neuron disease in mutant SOD1 transgenic mice. J Neurochem 2003; 87:752 - 63; http://dx.doi.org/10.1046/j.1471-4159.2003.02053.x; PMID: 14535957
   PubMed Web of Science ®Google Scholar
• McNeer NA, Chin JY, Schleifman EB, Fields RJ, Glazer PM, Saltzman WM. Nanoparticles deliver triplex-forming PNAs for site-specific genomic recombination in CD34+ human hematopoietic progenitors. Mol Ther 2011; 19:172 - 80; http://dx.doi.org/10.1038/mt.2010.200; PMID: 20859257
   PubMed Web of Science ®Google Scholar