Cell-type-specific aptamer and aptamer-small interfering RNA conjugates for targeted human immunodeficiency virus type 1 therapy

Journal of Investigative Medicine

Volumn 62, Issue 7, 2014, Pages 914-919

  Zhou, Jiehua ^a   Rossi, John ^a  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

Author keywords

Cell type specific siRNA delivery; HIV 1; HIV 1 gp120 aptamer; RNA interference; SiRNA

Indexed keywords

ANTIVIRUS AGENT; APTAMER; APTAMER SMALL INTERFERING RNA; CELL TYPE SPECIFIC APTAMER; RECOMBINANT GLYCOPROTEIN GP 120; SMALL INTERFERING RNA; UNCLASSIFIED DRUG; GLYCOPROTEIN GP 120;

ANTIVIRAL THERAPY; CHIMERA; CONFERENCE PAPER; CONJUGATE; DRUG TARGETING; GENE TARGETING; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS 1 INFECTION; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); RNA INTERFERENCE; VIRAL GENE DELIVERY SYSTEM; VIRUS REPLICATION; HIV INFECTIONS; METABOLISM; MOLECULARLY TARGETED THERAPY; PHYSIOLOGY; VIROLOGY;

APTAMERS, NUCLEOTIDE; HIV ENVELOPE PROTEIN GP120; HIV INFECTIONS; HIV-1; HUMANS; MOLECULAR TARGETED THERAPY; RNA, SMALL INTERFERING;

EID: 84925142753     PISSN: 17088267     EISSN: None     Source Type: Journal    
DOI: 10.1097/JIM.0000000000000103     Document Type: Conference Paper

References (71)

1. Pomerantz RJ, Horn DL. Twenty years of therapy for HIV-1 infection. Nat Med. 2003;9:867-873.
2. Richman DD, Margolis DM, Delaney M, et al. The challenge of finding a cure for HIV infection. Science. 2009;323:1304-1307.
3. Izzedine H, Harris M, Perazella MA. The nephrotoxic effects of HAART. Nat Rev Nephrol. 2009;5:563-573.
4. Joshi PJ, Fisher TS, Prasad VR. Anti-HIV inhibitors based on nucleic acids: emergence of aptamers as potent antivirals. Curr Drug Targets Infect Disord. 2003;3:383-400.
5. Scherer L, Rossi JJ, Weinberg MS. Progress and prospects: RNA-based therapies for treatment of HIV infection. Gene Ther. 2007;14:1057-1064.
6. Castanotto D, Rossi JJ, Sarver N. Antisense catalytic RNAs as therapeutic agents. Adv Pharmacol. 1994;25:289-317.
7. Chang HK, Gendelman R, Lisziewicz J, et al. Block of HIV-1 infection by a combination of antisense tat RNA and TAR decoys: a strategy for control of HIV-1. Gene Ther. 1994;1:208-216.
8. Michienzi A, Li S, Zaia JA, et al. A nucleolar TAR decoy inhibitor of HIV-1 replication. Proc Natl Acad Sci U S A. 2002;99:14047-14052.
9. Zaia JA, Rossi JJ, Murakawa GJ, et al. Inhibition of human immunodeficiency virus by using an oligonucleoside methylphosphonate targeted to the tat-3 gene. J Virol. 1988;62:3914-3917.
10. Zaia JA, Chatterjee S, Wong KK, et al. Status of ribozyme and antisense-based developmental approaches for anti-HIV-1 therapy. Ann N Y Acad Sci. 1992;660:95-106.
11. Rossi JJ, Sarver N. RNA enzymes (ribozymes) as antiviral therapeutic agents. Trends Biotechnol. 1990;8:179-183.
12. Sarver N, Cantin EM, Chang PS, et al. Ribozymes as potential anti-HIV-1 therapeutic agents. Science. 1990;247:1222-1225.
13. Dropulic B, Elkins DA, Rossi JJ, et al. Ribozymes: use as anti-HIV therapeutic molecules. Antisense Res Dev. 1993;3:87-94.
14. Rossi JJ, Cantin EM, Zaia JA, et al. Ribozymes as therapies for AIDS. Ann N Y Acad Sci. 1990;616:184-200.
15. Rossi JJ. The application of ribozymes to HIV infection. Curr Opin Mol Ther. 1999;1:316-322.
16. Rossi JJ. Ribozyme therapy for HIV infection. Adv Drug Deliv Rev. 2000;44:71-78.
17. Held DM, Kissel JD, Patterson JT, et al. HIV-1 inactivation by nucleic acid aptamers. Front Biosci. 2006;11:89-112.
18. Zhou J, Rossi JJ. Aptamer-targeted RNAi for HIV-1 therapy. Methods Mol Biol. 2011;721:355-371.
19. Zhou J, Rossi JJ. Progress in RNAi-based antiviral therapeutics. Methods Mol Biol 2011;721:67-75.
20. Novina CD, Murray MF, Dykxhoorn DM, et al. siRNA-directed inhibition of HIV-1 infection. Nat Med. 2002;8:681-686.
21. Lee NS, Rossi JJ. Control of HIV-1 replication by RNA interference. Virus Res. 2004;102:53-58.
22. Singh SK, Gaur RK. Progress towards therapeutic application of RNA interference for HIV infection. BioDrugs. 2009;23:269-276.
23. Goff SP. Knockdown screenstoknockout HIV-1. Cell. 2008;135:417-420.
24. Singh SK. RNA interference and its therapeutic potential against HIV infection. Expert Opin Biol Ther. 2008;8:449-461.
25. Zhou H, Xu M, Huang Q, et al. Genome-scale RNAi screen for host factors required for HIV replication. Cell Host Microbe. 2008;4:495-504.
26. Strayer DS, Akkina R, Bunnell BA, et al. Current status of gene therapy strategies to treat HIV/AIDS. Mol Ther. 2005;11:823-842.
27. Das AT, Brummelkamp TR, Westerhout EM, et al. Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition. J Virol. 2004;78:2601-2605.
28. Haasnoot J, Westerhout EM, Berkhout B. RNA interference against viruses: strike and counterstrike. Nat Biotechnol. 2007;25:1435-1443.
29. Boden D, Pusch O, Lee F, et al. Human immunodeficiency virus type 1 escape from RNA interference. J Virol. 2003;77:11531-11535.
30. ter Brake O, 't Hooft K, Liu YP, et al. Lentiviral vector design for multiple shRNA expression and durable HIV-1 inhibition. Mol Ther. 2008;16:557-564.
31. Li MJ, Kim J, Li S, et al. Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy. Mol Ther. 2005;12:900-909.
32. DiGiusto DL, Krishnan A, Li L, et al. RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma. Sci Transl Med. 2010;2:36ra43.
33. Sundaram P, Kurniawan H, Byrne ME, et al. Therapeutic RNA aptamers in clinical trials. Eur J Pharm Sci. 2013;48:259-271.
34. Ray P, Viles KD, Soule EE, et al. Application of aptamers for targeted therapeutics. Arch Immunol Ther Exp (Warsz). 2013;61:255-271.
35. Zhou J, Rossi JJ. Cell-specific aptamer-mediated targeted drug delivery. Oligonucleotides. 2011;21:1-10.
36. Zhou J, Rossi JJ. Therapeutic potential of Aptamer-siRNA conjugates for treatment of HIV-1. BioDrugs. 2012;26:393-400.
37. Li X, Zhao Q, Qiu L. Smart ligand: aptamer-mediated targeted delivery of chemotherapeutic drugs and siRNA for cancer therapy. J Control Release. 2013;171:152-162.
38. Zhou J, Li H, Li S, et al. Novel dual inhibitory function aptamer-siRNA delivery system for HIV-1 therapy. Mol Ther. 2008;16:1481-1489.
39. Zhou J, Swiderski P, Li H, et al. Selection, characterization and application of new RNA HIV gp 120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells. Nucleic Acids Res. 2009;37:3094-3109.
40. Kraus E, James W, Barclay AN. Cutting edge: novel RNA ligands able to bind CD4 antigen and inhibit CD4+ T lymphocyte function. J Immunol. 1998;160:5209-5212.
41. Wheeler LA, Trifonova R, Vrbanac V, et al. Inhibition of HIV transmission in human cervicovaginal explants and humanized mice using CD4 aptamer-siRNA chimeras. J Clin Invest. 2011;121:2401-2412.
42. Robertson DL, Joyce GF. Selection in vitro of an RNA enzyme that specifically cleaves single-stranded DNA. Nature. 1990;344:467-468.
43. Tuerk C, Gold L. Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase. Science. 1990;249:505-510.
44. Ellington AD, Szostak JW. In vitro selection of RNA molecules that bind specific ligands. Nature. 1990;346:818-822.
45. Kulbachinskiy AV. Methods for selection of aptamers to protein targets. Biochemistry (Mosc). 2007;72:1505-1518.
46. Guo KT, Paul A, Schichor C, et al. CELL-SELEX: novel perspectives of aptamer-based therapeutics. Int J Mol Sci. 2008;9:668-678.
47. Xiao Z, Shangguan D, Cao Z, et al. Cell-specific internalization study of an aptamer from whole cell selection. Chemistry. 2008;14:1769-1775.
48. Ohuchi S. Cell-SELEX technology. Biores Open Access. 2012;1:265-272.
49. Mi J, Liu Y, Rabbani ZN, et al. In vivo selection of tumor-targeting RNA motifs. Nat Chem Biol. 2010;6:22-24.
50. Cheng C, Chen YH, Lennox KA, et al. In vivo SELEX for identification of brain-penetrating aptamers. Mol Ther Nucleic Acids. 2013;2:e67.
51. Kwong PD, Wyatt R, Majeed S, et al. Structures of HIV-1 gp120 envelope glycoproteins from laboratory-adapted and primary isolates. Structure. 2000;8:1329-1339.
52. Kwong PD, Wyatt R, Robinson J, et al. Structure of an HIV gp120 envelope glycoprotein in complex with the CD4 receptor and a neutralizing human antibody. Nature. 1998;393:648-659.
53. Sattentau QJ, Moore JP. The role of CD4 in HIV binding and entry. Philos Trans R Soc Lond B Biol Sci. 1993;342:59-66.
54. Ugolini S, Mondor I, Sattentau QJ. HIV-1 attachment: another look. Trends Microbiol. 1999;7:144-149.
55. Ryser HJ, Fluckiger R. Progress in targeting HIV-1 entry. Drug Discov Today. 2005;10:1085-1094.
56. Sayer N, Ibrahim J, Turner K, et al. Structural characterization of a 2'F-RNA aptamer that binds a HIV-1 SU glycoprotein, gp120. Biochem Biophys Res Commun. 2002;293:924-931.
57. Khati M, Schuman M, Ibrahim J, et al. Neutralization of infectivity of diverse R5 clinical isolates of human immunodeficiency virus type 1 by gp120-binding 2'F-RNA aptamers. J Virol. 2003;77:12692-12698.
58. Dey AK, Griffiths C, Lea SM, et al. Structural characterization of an anti-gp120 RNA aptamer that neutralizes R5 strains of HIV-1. RNA. 2005;11:873-884.
59. Dey AK, Khati M, Tang M, et al. An aptamer that neutralizes R5 strains of human immunodeficiency virus type 1 blocks gp120-CCR5 interaction. J Virol. 2005;79:13806-13810.
60. Cohen C, Forzan M, Sproat B, et al. An aptamer that neutralizes R5 strains of HIV-1 binds to core residues of gp120 in the CCR5 binding site. Virology. 2008;381:46-54.
61. Zhou J, Swiderski P, Li H, et al. Selection, characterization and application of new RNA HIV gp 120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells. Nucleic Acids Res. 2009;37:3094-3109.
62. McNamara JO 2nd, Andrechek ER, Wang Y, et al. Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras. Nat Biotechnol. 2006;24:1005-1015.
63. Neff CP, Zhou J, Remling L, et al. An aptamer-siRNA chimera suppresses HIV-1 viral loads and protects from helper CD4(+) T cell decline in humanized mice. Sci Transl Med. 2011;3:66ra66.
64. Berges BK, Wheat WH, Palmer BE, et al. HIV-1 infection and CD4 T cell depletion in the humanized Rag2j/jgamma cj/j (RAG-hu) mouse model. Retrovirology. 2006;3:76.
65. Berges BK, Akkina SR, Folkvord JM, et al. Mucosal transmission of R5 and X4 tropic HIV-1 via vaginal and rectal routes in humanized Rag2j/jgammac j/j (RAG-hu) mice. Virology. 2008;373:342-351.
66. Berges BK, Akkina SR, Remling L, et al. Humanized Rag2(j/j)gammac(j/j) (RAG-hu) mice can sustain long-term chronic HIV-1 infection lasting more than a year. Virology. 2010;397:100-103.
67. Berges BK, Rowan MR. The utility of the new generation of humanized mice to study HIV-1 infection: transmission, prevention, pathogenesis, and treatment. Retrovirology. 2011;8:65.
68. Zhou J, Neff CP, Swiderski P, et al. Functional in vivo delivery of multiplexed anti-HIV-1 siRNAs via a chemically synthesized aptamer with a sticky bridge. Mol Ther. 2013;21:192-200.
69. Tucker CE, Chen LS, Judkins MB, et al. Detection and plasma pharmacokinetics of an anti-vascular endothelial growth factor oligonucleotide-aptamer (NX1838) in rhesus monkeys. J Chromatogr B Biomed Sci Appl. 1999;732:203-212.
70. Drolet DW, Nelson J, Tucker CE, et al. Pharmacokinetics and safety of an anti-vascular endothelial growth factor aptamer (NX1838) following injection into the vitreous humor of rhesus monkeys. Pharm Res. 2000;17:1503-1510.
71. Keefe AD, Cload ST. SELEX with modified nucleotides. Curr Opin Chem Biol. 2008;12:448-456.