全部 标题 作者
关键词 摘要

OALib Journal期刊
ISSN: 2333-9721
费用:99美元

查看量下载量

相关文章

更多...

Delivery of RNAi-Based Oligonucleotides by Electropermeabilization

DOI: 10.3390/ph6040510

Keywords: electroporation, siRNA, miRNA, LNA, siLNA

Full-Text   Cite this paper   Add to My Lib

Abstract:

For more than a decade, understanding of RNA interference (RNAi) has been a growing field of interest. The potent gene silencing ability that small oligonucleotides have offers new perspectives for cancer therapeutics. One of the present limits is that many biological barriers exist for their efficient delivery into target cells or tissues. Electropermeabilization (EP) is one of the physical methods successfully used to transfer small oligonucleotides into cells or tissues. EP consists in the direct application of calibrated electric pulses to cells or tissues that transiently permeabilize the plasma membranes, allowing efficient in vitro and in vivo . cytoplasmic delivery of exogenous molecules. The present review reports on the type of therapeutic RNAi-based oligonucleotides that can be electrotransferred, the mechanism(s) of their electrotransfer and the technical settings for pre-clinical purposes.

References

[1]  Bumcrot, D.; Manoharan, M.; Koteliansky, V.; Sah, D.W. RNAi therapeutics: A potential new class of pharmaceutical drugs. Nat. Chem. Biol. 2006, 2, 711–719, doi:10.1038/nchembio839.
[2]  Fire, A.; Xu, S.; Montgomery, M.K.; Kostas, S.A.; Driver, S.E.; Mello, C.C. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998, 391, 806–811, doi:10.1038/35888.
[3]  Lee, R.C.; Feinbaum, R.L.; Ambros, V. The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14. Cell 1993, 75, 843–854.
[4]  Tong, A.W.; Zhang, Y.A.; Nemunaitis, J. Small interfering RNA for experimental cancer therapy. Curr. Opin. Mol. Ther. 2005, 7, 114–124.
[5]  Bartel, D.P. MicroRNAs: Genomics, biogenesis, mechanism, and function. Cell 2004, 116, 281–297, doi:10.1016/S0092-8674(04)00045-5.
[6]  Carthew, R.W.; Sontheimer, E.J. Origins and mechanisms of miRNAs and siRNAs. Cell 2009, 136, 642–655, doi:10.1016/j.cell.2009.01.035.
[7]  Whitehead, K.A.; Langer, R.; Anderson, D.G. Knocking down barriers: Advances in siRNA delivery. Nat. Rev. Drug Discov. 2009, 8, 129–138, doi:10.1038/nrd2742.
[8]  Garzon, R.; Marcucci, G.; Croce, C.M. Targeting microRNAs in cancer: Rationale, strategies and challenges. Nat. Rev. Drug Discov. 2010, 9, 775–789, doi:10.1038/nrd3179.
[9]  Cemazar, M.; Golzio, M.; Escoffre, J.M.; Couderc, B.; Sersa, G.; Teissie, J. In vivo imaging of tumor growth after electrochemotherapy with cisplatin. Biochem. Biophys. Res. Commun. 2006, 348, 997–1002, doi:10.1016/j.bbrc.2006.07.132.
[10]  Cemazar, M.; Golzio, M.; Sersa, G.; Hojman, P.; Kranjc, S.; Mesojednik, S.; Rols, M.P.; Teissie, J. Control by pulse parameters of DNA electrotransfer into solid tumors in mice. Gene Ther. 2009, 16, 635–644, doi:10.1038/gt.2009.10.
[11]  Golzio, M.; Mazzolini, L.; Paganin-Gioanni, A.; Teissie, J. Targeted gene silencing into solid tumors with electrically mediated siRNA delivery. Methods Mol. Biol. 2009, 555, 15–27.
[12]  Coster, H.G. A quantitative analysis of the voltage-current relationships of fixed charge membranes and the associated property of “punch-through”. Biophys. J. 1965, 5, 669–686, doi:10.1016/S0006-3495(65)86745-5.
[13]  Teissie, J.; Golzio, M.; Rols, M.P. Mechanisms of cell membrane electropermeabilization: A minireview of our present (lack of ?) knowledge. Biochim. Biophys. Acta 2005, 1724, 270–280, doi:10.1016/j.bbagen.2005.05.006.
[14]  Hu, W.Y.; Myers, C.P.; Kilzer, J.M.; Pfaff, S.L.; Bushman, F.D. Inhibition of retroviral pathogenesis by RNA interference. Curr. Biol. 2002, 15, 1301–1311.
[15]  Calegari, F.; Haubensak, W.; Yang, D.; Huttner, W.B.; Buchholz, F. Tissue-specific RNA interference in postimplantation mouse embryos with endoribonuclease-prepared short interfering RNA. Proc. Natl. Acad. Sci. USA 2002, 22, 14236–14240.
[16]  Pekarik, V.; Bourikas, D.; Miglino, N.; Joset, P.; Preiswerk, S.; Stoeckli, E.T. Screening for gene function in chicken embryo using RNAi and electroporation. Nat. Biotechnol. 2003, 21, 93–96.
[17]  Golzio, M.; Mazzolini, L.; Ledoux, A.; Paganin, A.; Izard, M.; Hellaudais, L.; Bieth, A.; Pillaire, M.J.; Cazaux, C.; Hoffmann, J.S.; et al. In vivo gene silencing in solid tumors by targeted electrically mediated siRNA delivery. Gene Ther. 2007, 14, 752–759, doi:10.1038/sj.gt.3302920.
[18]  Lewis, D.L.; Hagstrom, J.E.; Loomis, A.G.; Wolff, J.A.; Herweijer, H. Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. Nat. Genet. 2002, 32, 107–108.
[19]  Matsuda, T.; Cepko, C.L. Electroporation and RNA interference in the rodent retina in vivo and in vitro. Proc. Natl. Acad. Sci. USA 2004, 101, 16–22, doi:10.1073/pnas.2235688100.
[20]  Paganin-Gioanni, A.; Bellard, E.; Couderc, B.; Teissie, J.; Golzio, M. Tracking in vitro and in vivo siRNA electrotransfer in tumor cells. J. RNAi Gene Silencing 2008, 4, 281–288.
[21]  Wells, D.J. Gene therapy progress and prospects: Electroporation and other physical methods. Gene Ther. 2004, 11, 1363–1369, doi:10.1038/sj.gt.3302337.
[22]  Heller, L.C.; Heller, R. In vivo electroporation for gene therapy. Hum. Gene Ther. 2006, 17, 890–897, doi:10.1089/hum.2006.17.890.
[23]  Mir, L.M.; Glass, L.F.; Sersa, G.; Teissie, J.; Domenge, C.; Miklavcic, D.; Jaroszeski, M.J.; Orlowski, S.; Reintgen, D.S.; Rudolf, Z.; et al. Effective treatment of cutaneous and subcutaneous malignant tumours by electrochemotherapy. Br. J. Cancer 1998, 77, 2336–2342, doi:10.1038/bjc.1998.388.
[24]  Sersa, G.; Stabuc, B.; Cemazar, M.; Miklavcic, D.; Rudolf, Z. Electrochemotherapy with cisplatin: the systemic antitumour effectiveness of cisplatin can be potentiated locally by the application of electric pulses in the treatment of malignant melanoma skin metastases. Melanoma Res. 2000, 10, 381–385, doi:10.1097/00008390-200008000-00010.
[25]  Gothelf, A.; Mir, L.M.; Gehl, J. Electrochemotherapy: results of cancer treatment using enhanced delivery of bleomycin by electroporation. Cancer Treat Rev. 2003, 29, 371–387, doi:10.1016/S0305-7372(03)00073-2.
[26]  Sersa, G.; Miklavcic, D.; Cemazar, M.; Rudolf, Z.; Pucihar, G.; Snoj, M. Electrochemotherapy in treatment of tumours. Eur. J. Surg. Oncol. 2008, 34, 232–240, doi:10.1016/j.ejso.2007.05.016.
[27]  Daud, A.I.; DeConti, R.C.; Andrews, S.; Urbas, P.; Riker, A.I.; Sondak, V.K.; Munster, P.N.; Sullivan, D.M.; Ugen, K.E.; Messina, J.L. Phase I trial of interleukin-12 plasmid electroporation in patients with metastatic melanoma. J. Clin. Oncol. 2008, 26, 5896–5903.
[28]  Bodles-Brakhop, A.M.; Heller, R.; Draghia-Akli, R. Electroporation for the delivery of DNA-based vaccines and immunotherapeutics: current clinical developments. Mol. Ther. 2009, 17, 585–592, doi:10.1038/mt.2009.5.
[29]  Mali, B.; Jarm, T.; Snoj, M.; Sersa, G.; Miklavcic, D. Antitumor effectiveness of electrochemotherapy: A systematic review and meta-analysis. Eur. J. Surg. Oncol. 2013, 39, 4–16, doi:10.1016/j.ejso.2012.08.016.
[30]  Mir, L.M.; Devauchelle, P.; Quintin-Colonna, F.; Delisle, F.; Doliger, S.; Fradelizi, D.; Belehradek, J., Jr.; Orlowski, S. First clinical trial of cat soft-tissue sarcomas treatment by electrochemotherapy. Br. J. Cancer 1997, 76, 1617–1622, doi:10.1038/bjc.1997.606.
[31]  Tozon, N.; Kodre, V.; Sersa, G.; Cemazar, M. Effective treatment of perianal tumors in dogs with electrochemotherapy. Anticancer Res. 2005, 25, 839–845.
[32]  Kodre, V.; Cemazar, M.; Pecar, J.; Sersa, G.; Cor, A.; Tozon, N. Electrochemotherapy compared to surgery for treatment of canine mast cell tumours. In Vivo 2009, 23, 55–62.
[33]  Rols, M.P.; Tamzali, Y.; Teissie, J. Electrochemotherapy of horses. A preliminary clinical report. Bioelectrochemistry 2002, 55, 101–105, doi:10.1016/S1567-5394(01)00156-6.
[34]  Aihara, H.; Miyazaki, J. Gene transfer into muscle by electroporation in vivo. Nat. Biotechnol. 1998, 16, 867–870.
[35]  Mir, L.M.; Bureau, M.F.; Gehl, J.; Rangara, R.; Rouy, D.; Caillaud, J.M.; Delaere, P.; Branellec, D.; Schwartz, B.; Scherman, D. High-efficiency gene transfer into skeletal muscle mediated by electric pulses. Proc. Natl. Acad. Sci. USA 1999, 96, 4262–4267, doi:10.1073/pnas.96.8.4262.
[36]  Dean, D.A. Nonviral gene transfer to skeletal, smooth, and cardiac muscle in living animals. Am. J. Physiol. Cell. Physiol. 2005, 289, C233–C245, doi:10.1152/ajpcell.00613.2004.
[37]  Heller, R.; Jaroszeski, M.; Atkin, A.; Moradpour, D.; Gilbert, R.; Wands, J.; Nicolau, C. In vivo gene electroinjection and expression in rat liver. FEBS Lett. 1996, 389, 225–228, doi:10.1016/0014-5793(96)00590-X.
[38]  Liu, F.; Huang, L. Electric gene transfer to the liver following systemic administration of plasmid DNA. Gene Ther. 2002, 9, 1116–1119, doi:10.1038/sj.gt.3301733.
[39]  Titomirov, A.V.; Sukharev, S.; Kistanova, E. In vivo electroporation and stable transformation of skin cells of newborn mice by plasmid DNA. Biochim. Biophys. Acta 1991, 1088, 131–134, doi:10.1016/0167-4781(91)90162-F.
[40]  Vandermeulen, G.; Staes, E.; Vanderhaeghen, M.L.; Bureau, M.F.; Scherman, D.; Preat, V. Optimisation of intradermal DNA electrotransfer for immunisation. J. Control Release 2007, 124, 81–87, doi:10.1016/j.jconrel.2007.08.010.
[41]  Tupin, E.; Poirier, B.; Bureau, M.F.; Khallou-Laschet, J.; Vranckx, R.; Caligiuri, G.; Gaston, A.T.; Duong van Huyen, J.P.; Scherman, D.; Bariety, J.; et al. Non-viral gene transfer of murine spleen cells achieved by in vivo electroporation. Gene Ther. 2003, 10, 569–579, doi:10.1038/sj.gt.3301914.
[42]  Isaka, Y.; Yamada, K.; Takabatake, Y.; Mizui, M.; Miura-Tsujie, M.; Ichimaru, N.; Yazawa, K.; Utsugi, R.; Okuyama, A.; Hori, M.; et al. Electroporation-mediated HGF gene transfection protected the kidney against graft injury. Gene Ther. 2005, 12, 815–820, doi:10.1038/sj.gt.3302478.
[43]  Saito, T.; Nakatsuji, N. Efficient gene transfer into the embryonic mouse brain using in vivo electroporation. Dev. Biol. 2001, 240, 237–246, doi:10.1006/dbio.2001.0439.
[44]  Khoury, M.; Bigey, P.; Louis-Plence, P.; Noel, D.; Rhinn, H.; Scherman, D.; Jorgensen, C.; Apparailly, F. A comparative study on intra-articular versus systemic gene electrotransfer in experimental arthritis. J. Gene Med. 2006, 8, 1027–1036, doi:10.1002/jgm.922.
[45]  Pavlin, D.; Cemazar, M.; C?r, A.; Sersa, G.; Pogacnik, A.; Tozon, N. Electrogene therapy with interleukin-12 in canine mast cell tumors. Radiol. Oncol. 2011, 45, 31–39.
[46]  Bureau, M.F.; Gehl, J.; Deleuze, V.; Mir, L.M.; Scherman, D. Importance of association between permeabilization and electrophoretic forces for intramuscular DNA electrotransfer. Biochim. Biophys. Acta 2000, 1474, 353–359, doi:10.1016/S0304-4165(00)00028-3.
[47]  Satkauskas, S.; Bureau, M.F.; Puc, M.; Mahfoudi, A.; Scherman, D.; Miklavcic, D.; Mir, L.M. Mechanisms of in vivo DNA electrotransfer: respective contributions of cell electropermeabilization and DNA electrophoresis. Mol. Ther. 2002, 5, 133–140, doi:10.1006/mthe.2002.0526.
[48]  Sel, D.; Golzio, M.; Pucihar, G.; Tamzali, Y.; Miklavcic, D.; Teissie, J. Non invasive contact electrodes for in vivo localized cutaneous electropulsation and associated drug and nucleic acid delivery. J. Control Release 2009, 134, 125–131, doi:10.1016/j.jconrel.2008.11.003.
[49]  Gehl, J.; Sorensen, T.H.; Nielsen, K.; Raskmark, P.; Nielsen, S.L.; Skovsgaard, T.; Mir, L.M. In vivo electroporation of skeletal muscle: Threshold, efficacy and relation to electric field distribution. Biochim. Biophys. Acta 1999, 1428, 233–240, doi:10.1016/S0304-4165(99)00094-X.
[50]  Golzio, M.; Mazzolini, L.; Moller, P.; Rols, M.P.; Teissie, J. Inhibition of gene expression in mice muscle by in vivo electrically mediated siRNA delivery. Gene Ther. 2005, 12, 246–251, doi:10.1038/sj.gt.3302405.
[51]  Spugnini, E.P.; Citro, G.; Porrello, A. Rational design of new electrodes for electrochemotherapy. J. Exp. Clin. Cancer Res. 2005, 24, 245–254.
[52]  Tjelle, T.E.; Salte, R.; Mathiesen, I.; Kjeken, R. A novel electroporation device for gene delivery in large animals and humans. Vaccine 2006, 24, 4667–4670, doi:10.1016/j.vaccine.2005.08.068.
[53]  Cemazar, M.; Golzio, M.; Sersa, G.; Rols, M.P.; Teissié, J. Electrically-assisted nucleic acids delivery to tissues in vivo: where do we stand? Curr. Pharm. Des. 2006, 12, 3817–3825, doi:10.2174/138161206778559740.
[54]  Golzio, M.; Teissie, J.; Rols, M.P. Direct visualization at the single-cell level of electrically mediated gene delivery. Proc. Natl. Acad. Sci. USA 2002, 99, 1292–1297, doi:10.1073/pnas.022646499.
[55]  Escoffre, J.M.; Portet, T.; Wasungu, L.; Teissie, J.; Dean, D.; Rols, M.P. What is (still not) known of the mechanism by which electroporation mediates gene transfer and expression in cells and tissues. Mol. Biotechnol. 2009, 41, 286–295, doi:10.1007/s12033-008-9121-0.
[56]  Puc, M.; Kotnik, T.; Mir, L.M.; Miklavcic, D. Quantitative model of small molecules uptake after in vitro cell electropermeabilization. Bioelectrochemistry 2003, 60, 1–10, doi:10.1016/S1567-5394(03)00021-5.
[57]  Chabot, S.; Orio, J.; Castanier, R.; Bellard, E.; Nielsen, S.J.; Golzio, M.; Teissié, J. LNA-based oligonucleotide electrotransfer for miRNA inhibition. Mol. Ther. 2012, 20, 1590–1598, doi:10.1038/mt.2012.95.
[58]  Castanotto, D.; Rossi, J.J. The promises and pitfalls of RNA-interference-based therapeutics. Nature 2009, 457, 426–433.
[59]  Sigoillot, F.D.; King, R.W. Vigilance and validation: Keys to success in RNAi screening. ACS Chem. Biol. 2011, 6, 47–60, doi:10.1021/cb100358f.
[60]  Heidel, J.D.; Hu, S.; Liu, X.F.; Triche, T.J.; Davis, M.E. Lack of interferon response in animals to naked siRNAs. Nat. Biotechnol. 2004, 22, 1579–1582.
[61]  Paganin-Gioanni, A.; Bellard, E.; Escoffre, J.M.; Rols, M.P.; Teissie, J.; Golzio, M. Direct visualization at the single-cell level of siRNA electrotransfer into cancer cells. Proc. Natl. Acad. Sci. USA 2011, 108, 10443–10447.
[62]  Kishida, T.; Asada, H.; Gojo, S.; Ohashi, S.; Shin-Ya, M.; Yasutomi, K.; Terauchi, R.; Takahashi, K.A.; Kubo, T.; Imanishi, J.; et al. Sequence-specific gene silencing in murine muscle induced by electroporation-mediated transfer of short interfering RNA. J. Gene Med. 2004, 6, 105–110, doi:10.1002/jgm.456.
[63]  Inoue, A.; Takahashi, K.A.; Mazda, O.; Terauchi, R.; Arai, Y.; Kishida, T.; Shin-Ya, M.; Asada, H.; Morihara, T.; Tonomura, H.; et al. Electro-transfer of small interfering RNA ameliorated arthritis in rats. Biochem. Biophys. Res. Commun. 2005, 336, 903–908, doi:10.1016/j.bbrc.2005.08.198.
[64]  Akaneya, Y.; Jiang, B.; Tsumoto, T. RNAi-induced gene silencing by local electroporation in targeting brain region. J. Neurophysiol. 2005, 93, 594–602, doi:10.1152/jn.00161.2004.
[65]  Takabatake, Y.; Isaka, Y.; Mizui, M.; Kawachi, H.; Shimizu, F.; Ito, T.; Hori, M.; Imai, E. Exploring RNA interference as a therapeutic strategy for renal disease. Gene Ther. 2005, 12, 965–973, doi:10.1038/sj.gt.3302480.
[66]  Broderick, K.E.; Chan, A.; Lin, F.; Shen, X.; Kichaev, G.; Khan, A.S.; Aubin, J.; Zimmermann, T.S.; Sardesai, N.Y. Optimized in vivo transfer of small interfering RNA targeting dermal tissue using in vivo surface electroporation. Mol. Ther. Nucleic Acids 2012, 14, e11.
[67]  Nakai, N.; Kishida, T.; Shin-Ya, M.; Imanishi, J.; Ueda, Y.; Kishimoto, S.; Mazda, O. Therapeutic RNA interference of malignant melanoma by electrotransfer of small interfering RNA targeting Mitf. Gene Ther. 2007, 14, 357–365, doi:10.1038/sj.gt.3302868.
[68]  McCaffrey, A.P.; Meuse, L.; Pham, T.T.; Conklin, D.S.; Hannon, G.J.; Kay, M.A. RNA interference in adult mice. Nature 2002, 418, 38–39.
[69]  Jackson, A.L.; Linsley, P.S. Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application. Nat. Rev. Drug Discov. 2010, 9, 57–67, doi:10.1038/nrd3010.
[70]  Ambros, V. MicroRNAs: tiny regulators with great potential. Cell 2001, 107, 823–826, doi:10.1016/S0092-8674(01)00616-X.
[71]  Lu, J.; Getz, G.; Miska, E.A.; Alvarez-Saavedra, E.; Lamb, J.; Peck, D.; Sweet-Cordero, A.; Ebert, B.L.; Mak, R.H.; Ferrando, A.A.; et al. MicroRNA expression profiles classify human cancers. Nature 2005, 435, 834–838.
[72]  Hernando, E. MicroRNAs and cancer: Role in tumorigenesis, patient classification and therapy. Clin. Transl. Oncol. 2007, 9, 155–160, doi:10.1007/s12094-007-0029-0.
[73]  Da Costa Martins, P.A.; Salic, K.; Gladka, M.M.; Armand, A.S.; Leptidis, S.; El Azzouzi, H.; Hansen, A.; Coenen-de Roo, C.J.; Bierhuizen, M.F.; van der Nagel, R.; et al. MicroRNA-199b targets the nuclear kinase Dyrk1a in an auto-amplification loop promoting calcineurin/NFAT signalling. Nat. Cell. Biol. 2010, 12, 1220–1227, doi:10.1038/ncb2126.
[74]  Long, J.; Wang, Y.; Wang, W.; Chang, B.H.; Danesh, F.R. Identification of microRNA-93 as a novel regulator of vascular endothelial growth factor in hyperglycemic conditions. J. Biol. Chem. 2010, 285, 23457–23465.
[75]  Brodersen, P.; Voinnet, O. Revisiting the principles of microRNA target recognition and mode of action. Nat. Rev. Mol. Cell. Biol. 2009, 10, 141–148, doi:10.1038/nrm2619.
[76]  Stenvang, J.; Kauppinen, S. MicroRNAs as targets for antisense-based therapeutics. Expert Opin. Biol. Ther. 2008, 8, 59–81, doi:10.1517/14712598.8.1.59.
[77]  Stenvang, J.; Lindow, M.; Kauppinen, S. Targeting of microRNAs for therapeutics. Biochem. Soc. Trans. 2008, 36, 1197–1200, doi:10.1042/BST0361197.
[78]  Dalmay, T. MicroRNAs and cancer. J. Intern. Med. 2008, 263, 366–375, doi:10.1111/j.1365-2796.2008.01926.x.
[79]  Care, A.; Catalucci, D.; Felicetti, F.; Bonci, D.; Addario, A.; Gallo, P.; Bang, M.L.; Segnalini, P.; Gu, Y.; Dalton, N.D.; et al. MicroRNA-133 controls cardiac hypertrophy. Nat. Med. 2007, 13, 613–618.
[80]  Mercatelli, N.; Coppola, V.; Bonci, D.; Miele, F.; Costantini, A.; Guadagnoli, M.; Bonanno, E.; Muto, G.; Frajese, G.V.; de Maria, R.; et al. The inhibition of the highly expressed miR-221 and miR-222 impairs the growth of prostate carcinoma xenografts in mice. PLoS One 2008, 3, e4029.
[81]  Clape, C.; Fritz, V.; Henriquet, C.; Apparailly, F.; Fernandez, P.L.; Iborra, F.; Avances, C.; Villalba, M.; Culine, S.; Fajas, L. miR-143 interferes with ERK5 signaling, and abrogates prostate cancer progression in mice. PLoS One 2009, 4, e7542.
[82]  Layzer, J.M.; McCaffrey, A.P.; Tanner, A.K.; Huang, Z.; Kay, M.A.; Sullenger, B.A. In vivo activity of nuclease-resistant siRNAs. RNA 2004, 10, 766–771, doi:10.1261/rna.5239604.
[83]  Raemdonck, K.; Remaut, K.; Lucas, B.; Sanders, N.N.; Demeester, J.; De Smedt, S.C. In situ analysis of single-stranded and duplex siRNA integrity in living cells. Biochemistry 2006, 45, 10614–10623, doi:10.1021/bi060351b.
[84]  Corey, D.R. Chemical modification: The key to clinical application of RNA interference? J. Clin. Invest. 2007, 117, 3615–3622, doi:10.1172/JCI33483.
[85]  Braasch, D.A.; Jensen, S.; Liu, Y.; Kaur, K.; Arar, K.; White, M.A.; Corey, D.R. RNA interference in mammalian cells by chemically-modified RNA. Biochemistry 2003, 42, 7967–7975.
[86]  Kumar, R.; Singh, S.K.; Koshkin, A.A.; Rajwanshi, V.K.; Meldgaard, M.; Wengel, J. The first analogues of LNA (locked nucleic acids): Phosphorothioate-LNA and 2'-thio-LNA. Bioorg. Med. Chem. Lett. 1998, 8, 2219–2222, doi:10.1016/S0960-894X(98)00366-7.
[87]  Petersen, M.; Nielsen, C.B.; Nielsen, K.E.; Jensen, G.A.; Bondensgaard, K.; Singh, S.K.; Rajwanshi, V.K.; Koshkin, A.A.; Dahl, B.M.; Wengel, J.; et al. The conformations of locked nucleic acids (LNA). J. Mol. Recognit. 2000, 13, 44–53, doi:10.1002/(SICI)1099-1352(200001/02)13:1<44::AID-JMR486>3.0.CO;2-6.
[88]  Kaur, H.; Babu, B.R.; Mait, S. Perspectives on chemistry and therapeutic applications of Locked Nucleic Acid (LNA). Chem. Rev. 2007, 107, 4672–4697, doi:10.1021/cr050266u.
[89]  Crinelli, R.; Bianchi, M.; Gentilini, L.; Magnani, M. Design and characterization of decoy oligonucleotides containing locked nucleic acids. Nucleic Acids Res. 2002, 30, 2435–2443, doi:10.1093/nar/30.11.2435.
[90]  Wahlestedt, C.; Salmi, P.; Good, L.; Kela, J.; Johnsson, T.; Hokfelt, T.; Broberger, C.; Porreca, F.; Lai, J.; Ren, K.; et al. Potent and nontoxic antisense oligonucleotides containing locked nucleic acids. Proc. Natl. Acad. Sci. USA 2000, 97, 5633–5638, doi:10.1073/pnas.97.10.5633.
[91]  Lanford, R.E.; Hildebrandt-Eriksen, E.S.; Petri, A.; Persson, R.; Lindow, M.; Munk, M.E.; Kauppinen, S.; Orum, H. Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science 2010, 327, 198–201, doi:10.1126/science.1178178.
[92]  Braasch, D.A.; Liu, Y.; Corey, D.R. Antisense inhibition of gene expression in cells by oligonucleotides incorporating locked nucleic acids: Effect of mRNA target sequence and chimera design. Nucleic Acids Res. 2002, 30, 5160–5167, doi:10.1093/nar/gkf651.
[93]  Corsten, M.F.; Miranda, R.; Kasmieh, R.; Krichevsky, A.M.; Weissleder, R.; Shah, K. MicroRNA-21 knockdown disrupts glioma growth in vivo and displays synergistic cytotoxicity with neural precursor cell delivered S-TRAIL in human gliomas. Cancer Res. 2007, 67, 8994–9000, doi:10.1158/0008-5472.CAN-07-1045.
[94]  Pelofy, S.; Teissié, J.; Golzio, M.; Chabot, S. Chemically modified oligonucleotide-increased stability negatively correlates with its efficacy despite efficient electrotransfer. J. Membr. Biol. 2012, 245, 565–571, doi:10.1007/s00232-012-9468-9.

Full-Text

Contact Us

[email protected]

QQ:3279437679

WhatsApp +8615387084133