Emerging RNA-based Drugs: siRNAs, microRNAs and derivates

Central Nervous System Agents in Medicinal Chemistry

Volumn 12, Issue 3, 2012, Pages 217-232

  Pereira, Tiago Campos ^a   Lopes Cendes, Iscia ^b  

^a UNIVERSITY OF SÃO PAULO   (Brazil)

^b UNIVERSITY OF CAMPINAS   (Brazil)

Author keywords

Anti miRs; dsRNA; microRNA; RNA drugs; RNA interference; siRNA

Indexed keywords

ALPHA SYNUCLEIN; BETA SECRETASE 1; CATHEPSIN B; CD81 ANTIGEN; COPPER ZINC SUPEROXIDE DISMUTASE; ESTROGEN RECEPTOR ALPHA; GLUTAMATE DECARBOXYLASE 67; HUNTINGTIN; LEUCINE RICH REPEAT KINASE 2; MICRORNA; MU OPIATE RECEPTOR; PRION PROTEIN; SEROTONIN TRANSPORTER; SEROTONIN UPTAKE INHIBITOR; SIRTUIN 2; SMALL INTERFERING RNA; UROKINASE;

AMYOTROPHIC LATERAL SCLEROSIS; BRAIN MALARIA; CANDIDIASIS; CLINICAL PRACTICE; DRUG DELIVERY SYSTEM; DRUG HALF LIFE; GENE CONTROL; GENE SILENCING; GENETIC DISORDER; HAEMOPHILUS MENINGITIS; HALF LIFE TIME; HUMAN; INFECTION; MENINGOCOCCOSIS; NEOPLASM; NEUROCYSTICERCOSIS; NEUROSCHISTOSOMIASIS; NONHUMAN; PARASITOSIS; PHYSICIAN; POSTTRANSCRIPTIONAL GENE SILENCING; PRION DISEASE; REVIEW; SEXUAL BEHAVIOR; TOXOPLASMOSIS; TRYPANOSOMIASIS;

ANIMALS; DRUG DISCOVERY; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HUMANS; MENTAL DISORDERS; MICRORNAS; NERVOUS SYSTEM DISEASES; PHARMACEUTICAL PREPARATIONS; RNA, SMALL INTERFERING;

EID: 84866651259     PISSN: 18715249     EISSN: 18756166     Source Type: Journal    
DOI: 10.2174/187152412802430138     Document Type: Review

References (166)

1. Napoli, C.; Lemieux, C.; Jorgensen, R. Introduction of a chimeric chalcone synthase gene into petunia results in reversible cosuppression of homologous genes in trans. Plant Cell, 1990, 2(4), 279-289.
2. Wightman, B.; Ha, I.; Ruvkun, G. Posttranscriptional regulation of the heterochronic gene lin-14 by lin-4 mediates temporal pattern formation in C. elegans. Cell, 1993, 75(5), 855-862.
3. Meister. G.; Tuschl, T. Mechanisms of gene silencing by doublestranded RNA. Nature, 2004, 431, 343-349.
4. Elbashir, S.M.; Harborth, J.; Lendeckel, W.; Yalcin, A.; Weber, K.; Tuschl, T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature, 2001, 411, 494-498.
5. Holen, T.; Amarzguioui, M.; Wiiger, M.T.; Babaie, E.; Prydz, H. Positional effects of short interfering RNAs targeting the human coagulation trigger Tissue Factor. Nucleic Acids Res., 2002, 30, 1757-1766.
6. Khvorova, A.; Reynolds, A.; Jayasena, S.D. Functional siRNAs and miRNAs exhibit strand bias. Cell, 2003, 115, 209-216.
7. Reynolds, A.; Leake, D.; Boese, Q.; Scaringe, S.; Marshall, W.S.; Khvorova, A. Rational siRNA design for RNA interference. Nat. Biotechnol., 2004, 22, 326-330.
8. 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.
9. Schiffelers, R.M.; Ansari, A.; Xu, J.; Zhou, Q.; Tang, Q.; Storm, G.; Molema, G.; Lu, P.Y.; Scaria, P.V.; Woodle, M.C. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic Acids Res., 2004, 32, e149.
10. Soutschek, J.; Akinc, A.; Bramlage, B.; Charisse, K.; Constien, R.; Donoghue, M.; Elbashir, S.; Geick, A.; Hadwiger, P.; Harborth, J.; John, M.; Kesavan, V.; Lavine, G.; Pandey, RK.; Racie, T.; Rajeev, K.G.; Röhl, I.; Toudjarska, I.; Wang, G.; Wuschko, S.; Bumcrot, D.; Koteliansky, V.; Limmer, S.; Manoharan, M.; Vornlocher, H.P. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature, 2004, 432, 173-178.
11. Schröfelbauer B.; Hakata Y.; Landau NR. HIV-1 Vpr function is mediated by interaction with the damage-specific DNA-binding protein DDB1. Proc. Natl. Acad. Sci. U.S.A., 2007, 104, 4130-4135.
12. Fluiter. K.; Mook, O.R.; Baas, F. The therapeutic potential of LNA-modified siRNAs: reduction of off-target effects by chemical modification of the siRNA sequence. Methods Mol. Biol., 2009, 487, 189-203.
13. Morrissey, D.V.; Lockridge, J.A.; Shaw, L.; Blanchard, K.; Jensen, K.; Breen, W.; Hartsough, K.; Machemer. L.; Radka. S.; Jadhav. V.; Vaish. N.; Zinnen. S.; Vargeese. C.; Bowman. K.; Shaffer. C.S.; Jeffs, L.B.; Judge, A.; MacLachlan, I.; Polisky, B. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nature Biotechnol., 2005, 23, 1002-1007.
14. Robbins, M.; Judge, A.; Liang, L.; McClintock, K.; Yaworski, E.; MacLachlan, I. 2'-O-Methyl-modified RNAs act as TLR7 antagonists. Mol. Ther., 2007, 15, 1663-1669.
15. Fisher, M.; Abramov, M.; Van Aerschot, A.; Xu, D.; Juliano, R.L.; Herdewijn, P. Inhibition of MDR1 expression with altritolmodified siRNAs. Nucleic Acids. Res., 2007, 35, 1064-1074.
16. Dowler, T.; Bergeron, D.; Tedeschi, A.L.; Paquet, L.; Ferrari, N.; Damha, M.J. Improvements in siRNA properties mediated by 2'-deoxy-2'-fluoro-μ-d-arabinonucleic acid (FANA). Nucleic Acids Res., 2006, 34, 1669-1675.
17. Watts, J.K.; Choubdar, N.; Sadalapure, K.; Robert, F.; Wahba, A.S.; Pelletier, J.; Pinto, B.M.; Damha, M.J. 2'-Fluoro-4'-thioarabino-modified oligonucleotides: conformational switches linked to siRNA activity. Nucleic Acids Res., 2007, 35, 1441-1451.
18. Gaglione, M.; Messere, A. Recent progress in chemically modified siRNAs. Mini Rev. Med. Chem., 2010, 10 (7), 578-595.
19. Brummelkamp, T.R.; Bernards, R.; Agami, R. A system for stable expression of short interfering RNAs in mammalian cells. Science, 2002, 296, 550-553.
20. Hohjoh, H. Enhancement of RNAi activity by improved siRNA duplexes. FEBS Lett., 2004, 557, 193-198.
21. Kim, D.H.; Behlke, M.A.; Rose, S.D.; Chang, M.S.; Choi, S.; Rossi, J.J. Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy. Nat. Biotechnol., 2005, 23, 222-226.
22. Yang, D.; Buchholz, F.; Huang, Z.; Goga, A.; Chen, C.Y.; Brodsky, F.M.;, Bishop, J.M. Short RNA duplexes produced by hydrolysis with Escherichia coli RNase III mediate effective RNA interference in mammalian cells. Proc. Natl. Acad. Sci. U.S.A., 2002, 99, 9942-9947.
23. Sun, X.; Rogoff, H.A.; Li, C.J. Asymmetric RNA duplexes mediate RNA interference in mammalian cells. Nat. Biotechnol., 2008, 26 (12), 1379-1382.
24. Chu, C.Y.; Rana, T.M. Potent RNAi by short RNA triggers. RNA, 2008, 14 (9), 1714-1719.
25. Bolcato-Bellemin, A.L.; Bonnet, M.E.; Creusat, G.; Erbacher, P.; Behr, J.P. Sticky overhangs enhance siRNA-mediated gene silencing. Proc. Natl. Acad. Sci. U.S.A., 2007, 104 (41), 16050-16055.
26. Seyhan AA, Vlassov AV, Johnston BH. RNA interference from multimeric shRNAs generated by rolling circle transcription. Oligonucleotides, 2006, 16(4), 353-363.
27. Caplen, N.J.; Taylor, J.P.; Statham, V.S.; Tanaka, F.; Fire, A.; Morgan, R.A. Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interference. Hum. Mol. Genet., 2002, 11, 175-184.
28. Koldehoff, M.; Steckel, N.K.; Beelen, D.W.; Elmaagacli, A.H. Therapeutic application of small interfering RNA directed against bcr-abl transcripts to a patient with imatinib-resistant chronic myeloid leukaemia. Clin. Exp. Med., 2007, 7(2), 47-55.
29. Koldehoff, M.; Elmaagacli, A.H. Therapeutic targeting of gene expression by siRNAs directed against BCR-ABL transcripts in a patient with imatinib-resistant chronic myeloid leukemia. Methods Mol. Biol., 2009, 487, 451-466.
30. Davis, M.E. The first targeted delivery of siRNA in humans via a self-assembling, cyclodextrin polymer-based nanoparticle: from concept to clinic. Mol. Pharm., 2009, 6 (3), 659-668.
31. Leachman, S. A,; Hickerson, R.P.; Schwartz, M.E.; Bullough, E.E.; Hutcherson, S.L.; Boucher, K.M.; Hansen, C.D.; Eliason, M.J.; Srivatsa, G.S.; Kornbrust, D.J.; Smith, F.J.; McLean, W.I.; Milstone, L.M.; Kaspar, R.L. First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder. Mol. Ther., 2010, 18 (2), 442-446.
32. Davis, M.E.; Zuckerman, J.E.; Choi. C.H.; Seligson, D.; Tolcher, A.; Alabi, C.A.; Yen, Y.; Heidel, J.D.; Ribas, A. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature, 2010, 464, 1067-1070.
33. Bahi, A.; Boyer, F.; Kolira, M.; Dreyer, J.L. In vivo gene silencing of CD81 by lentiviral expression of small interference RNAs suppresses cocaine-induced behaviour. J. Neurochem., 2005, 92, 1243-1255.
34. Pfeifer, A.; Eigenbrod, S.; Al-Khadra, S.; Hofmann, A.; Mitteregger, G.; Moser, M.; Bertsch, U.; Kretzschmar, H. Lentivector-mediated RNAi efficiently suppresses prion protein and prolongs survival of scrapie-infected mice. J. Clin. Invest., 2006, 116, 3204-3210.
35. Carmell, M.A.; Zhang, L.; Conklin, D.S.; Hannon, G.J.; Rosenquist, T.A. Germline transmission of RNAi in mice. Nat. Struct. Biol., 2003, 10, 91-92.
36. Chen, Q.; Butler, D.; Querbes, W.; Pandey, R.K.; Ge, P.; Maier, M.A.; Zhang, L.; Rajeev, K.G.; Nechev, L.; Kotelianski, V.; Manoharan, M.; Sah. D.W. Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery. J. Control Release, 2010, 144, 227-232.
37. Yang, T.; Choi, M.K.; Cui, F.D.; Lee, S.J.; Chung, S.J.; Shim, C.K.; Kim, D.D. Antitumor effect of paclitaxel-loaded PEGylated immunoliposomes against human breast cancer cells. Pharm. Res,. 2007, 24, 2402-2411.
38. Boado R.J. Blood-brain barrier transport of non-viral gene and RNAi therapeutics. Pharm. Res., 2007, 24, 1772-1787.
39. Zhang, Y.; Zhang, Y.F.; Bryant, J.; Charles, A.; Boado, R.J.; Pardridge, W.M. Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer. Clin. Cancer Res., 2004, 10, 3667-3677.
40. Kumar, P.; Wu, H.; McBride, J.L.; Jung, K.E.; Kim, M.H.; Davidson, B.L.; Lee, S.K.; Shankar, P.; Manjunath, N. Transvascular delivery of small interfering RNA to the central nervous system. Nature, 2007, 448, 39-43.
41. Alvarez-Erviti L, Seow Y, Yin H, Betts C, Lakhal S, Wood MJ. Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat. Biotechnol., 2011, 29, 341-345
42. Castilla, J.; Saá, P.; Soto, C. Detection of prions in blood. Nat. Med., 2005, 11, 982-985.
43. Daude, N.; Marella, M.; Chabry, J. Specific inhibition of pathological prion protein accumulation by small interfering RNAs. J. Cell Sci., 2003, 116, 2775-2779.
44. Atarashi, R.; Moore, R.A.; Sim, V.L.; Hughson, A.G.; Dorward D.W.; Onwubiko H.A.; Priola S.A.; Caughey B. Ultrasensitive detection of scrapie prion protein using seeded conversion of recombinant prion protein. Nat. Methods, 2007, 4, 645-650.
45. Pulford, B.; Reim, N.; Bell, A.; Veatch, J.; Forster, G.; Bender, H.; Meyerett, C.; Hafeman, S.; Michel, B.; Johnson, T.; Wyckoff, A.C.; Miele, G.; Julius, C.; Kranich, J.; Schenkel, A.; Dow, S.; Zabel, M.D. Liposome-siRNA-peptide complexes cross the bloodbrain barrier and significantly decrease PrP on neuronal cells and PrP in infected cell cultures. PLoS One, 2010, 5, e11085.
46. Leucht, C.; Simoneau, S.; Rey, C.; Vana, K.; Rieger, R.; Lasmézas, CI.; Weiss, S. The 37 kDa/67 kDa laminin receptor is required for PrP(Sc) propagation in scrapie-infected neuronal cells. EMBO Rep., 2003, 4, 290-295.
47. Pflanz, H.; Vana, K.; Mitteregger, G.; Pace, C.; Messow, D.; Sedlaczek, C.; Nikles, D.; Kretzschmar, H.A.; Weiss, S.F. Microinjection of lentiviral vectors expressing small interfering RNAs directed against laminin receptor precursor mRNA prolongs the pre-clinical phase in scrapie-infected mice. J. Gen. Virol., 2009, 90, 269-274.
48. Ludewigs, H.; Zuber, C.; Vana, K.; Nikles, D.; Zerr, I.; Weiss, S. Therapeutic approaches for prion disorders. Expert Rev. Anti Infect. Ther., 2007, 5, 613-630.
49. Zafar, S.; von Ahsen, N.; Oellerich, M.; Zerr, I.; Schulz-Schaeffer, W.J.; Armstrong, V.W.; Asif, A.R. Proteomics approach to identify the interacting partners of cellular prion protein and characterization of Rab7a interaction in neuronal cells. J. Proteome Res., 2011, 10, 3123-3135.
50. Jacque, JM.; Triques, K.; Stevenson, M. Modulation of HIV-1 replication by RNA interference. Nature, 2002, 418, 435-438.
51. Wiebusch, L.; Truss, M.; Hagemeier, C. Inhibition of human cytomegalovirus replication by small interfering RNAs. J. Gen. Virol. 2004, 85, 179-184.
52. Palliser, D.; Chowdhury, D.; Wang, Q.Y.; Lee, SJ.; Bronson, R.T.; Knipe, D.M.; Lieberman, J. An siRNA-based microbicide protects mice from lethal herpes simplex virus 2 infection. Nature, 2006, 439, 89-94.
53. Gitlin, L.; Karelsky, S.; Andino, R. Short interfering RNA confers intracellular antiviral immunity in human cells. Nature, 2002, 418, 430-434.
54. Novina, C.D.; Murray, M.F.; Dykxhoorn, D.M.; Beresford, P.J.; Riess, J.; Lee, S.K.; Collman, R.G.; Lieberman, J.; Shankar, P.; Sharp, P.A. siRNA-directed inhibition of HIV-1 infection. Nat. Med., 2002, 8, 681-686.
55. Arteaga H.J.; Hinkula J.; van Dijk-Härd, I.; Dilber, M.S.; Wahren, B.; Christensson, B.; Mohamed, A.J.; Smith, C.I. Choosing CCR5 or Rev siRNA in HIV-1. Nat. Biotechnol., 2003, 21, 230-231.
56. Ryo, A.; Tsurutani, N.; Ohba, K.; Kimura, R.; Komano, J.; Nishi, M.; Soeda, H.; Hattori, S.; Perrem, K.; Yamamoto, M.; Chiba, J.; Mimaya, J.; Yoshimura, K.; Matsushita, S.; Honda, M.; Yoshimura, A.; Sawasaki, T.; Aoki, I.; Morikawa, Y.; Yamamoto, N. SOCS1 is an inducible host factor during HIV-1 infection and regulates the intracellular trafficking and stability of HIV-1 Gag. Proc. Natl. Acad. Sci. U.S.A., 2008, 105, 294-299.
57. Bortolamiol, D.; Pazhouhandeh, M.; Marrocco, K.; Genschik, P.; Ziegler-Graff, V. The Polerovirus F box protein P0 targets ARGONAUTE1 to suppress RNA silencing. Curr. Biol., 2007, 17, 1615-1621.
58. Cullen, B.R. Is RNA interference involved in intrinsic antiviral immunity in mammals? Nat. Immunol., 2006, 7, 563-567.
59. Bivalkar-Mehla, S.; Vakharia, J.; Mehla, R.; Abreha, M.; Kanwar, J.R.; Tikoo, A.; Chauhan, A. Viral RNA silencing suppressors (RSS): novel strategy of viruses to ablate the host RNA interference (RNAi) defense system. Virus Res., 2011, 155:1-9.
60. Churikov, N.A.; Chistiakova, L.G.; Zavil'gel'ski, G.B.; Manukhov, I.V. [RNA interference in Escherichia coli cells: the expression of molecules that are complementary to the lon gene mRNA in parallel orientation]. In Russian. Genetika, 2000, 36, 23-27.
61. Yanagihara, K.; Tashiro, M.; Fukuda, Y.; Ohno, H.; Higashiyama, Y.; Miyazaki, Y.; Hirakata, Y.; Tomono, K.; Mizuta, Y.; Tsukamoto, K.; Kohno, S. Effects of short interfering RNA against methicillin-resistant Staphylococcus aureus coagulase in vitro and in vivo. J. Antimicrob. Chemother., 2006, 57, 122-126.
62. Schubert-Unkmeir, A.; Konrad, C.; Slanina, H.; Czapek, F.; Hebling, S.; Frosch, M. Neisseria meningitidis induces brain microvascular endothelial cell detachment from the matrix and cleavage of occludin: a role for MMP-8. PLoS Pathog., 2010, 6, e1000874.
63. Long, M.; Huang, S.H.; Wu, C.H.; Shackleford, G.M.; Jong, A. Lipid raft/caveolae signaling is required for Cryptococcus neoformans invasion into human brain microvascular endothelial cells. J. Biomed. Sci., 2012, 19, 19.
64. Huang, S.H.; Long, M.; Wu, C.H.; Kwon-Chung, K.J.; Chan, Y.C.; Chi, F.; Lee, S.; Jong, A. Invasion of Cryptococcus neoformans into human brain microvascular endothelial cells is mediated through the lipid rafts-endocytic pathway via the dual specificity tyrosine phosphorylation-regulated kinase 3 (DYRK3). J. Biol. Chem., 2011, 286, 34761-34769.
65. Chen, Y.; Chen, J.; Wen, H.; Gao, P.; Wang. J.; Zheng. Z.; Gu. J. S100A10 downregulation inhibits the phagocytosis of Cryptococcus neoformans by murine brain microvascular endothelial cells. Microb. Pathog., 2011, 51, 96-100.
66. Qin, Q.M.; Luo, J.; Lin, X.; Pei, J.; Li, L.; Ficht, T.A.; de Figueiredo, P. Functional analysis of host factors that mediate the intracellular lifestyle of Cryptococcus neoformans. PLoS Pathog., 2011, 7,:e1002078.
67. Karlas, A.; Machuy, N.; Shin, Y.; Pleissner, K.P.; Artarini, A.; Heuer, D.; Becker, D.; Khalil, H.; Ogilvie, L.A.; Hess, S.; Mäurer, A.P.; Müller, E.; Wolff, T.; Rudel, T.; Meyer, T.F. Genome-wide RNAi screen identifies human host factors crucial for influenza virus replication. Nature, 2010, 463, 818-822.
68. Cronin, S. J; Nehme, N.T.; Limmer, S.; Liegeois, S.; Pospisilik, J.A.; Schramek, D.; Leibbrandt, A.; Simoes, R, de M.; Gruber, S.; Puc, U.; Ebersberger, I.; Zoranovic, T.; Neely, G.G.; von Haeseler, A.; Ferrandon, D.; Penninger, J.M. Genome-wide RNAi screen identifies genes involved in intestinal pathogenic bacterial infection. Science, 2009, 325, 340-343.
69. Kumar, D.; Nath, L.; Kamal, M.A.; Varshney, A.; Jain, A.; Singh, S.; Rao, K.V. Genome-wide analysis of the host intracellular network that regulates survival of Mycobacterium tuberculosis. Cell., 2010, 140, 731-743.
70. Abdulla, M.H.; O'Brien, T.; Mackey, Z.B.; Sajid, M.; Grab, D.J.; McKerrow, J.H. RNA interference of Trypanosoma brucei cathepsin B and L affects disease progression in a mouse model. PLoS Negl. Trop. Dis., 2008, 2, e298.
71. Pereira, T.C.; Pascoal, V.D.; Marchesini, R.B.; Maia, I.G.; Magalhães, L.A.; Zanotti-Magalhães, E.M.; Lopes-Cendes, I. Schistosoma mansoni: evaluation of an RNAi-based treatment targeting HGPRTase gene. Exp. Parasitol., 2008, 118, 619.
72. Chandramohanadas. R.; Davis, P.H.; Beiting, D.P.; Harbut, M.B.; Darling, C.; Velmourougane, G.; Lee, M.Y.; Greer, P.A.; Roos, D.S.; Greenbaum, D.C. Apicomplexan parasites co-opt host calpains to facilitate their escape from infected cells. Science, 2009, 324, 794-797.
73. Kasinathan, R.S.; Morgan, W.M.; Greenberg, R.M. Genetic knockdown and pharmacological inhibition of parasite multidrug resistance transporters disrupts egg production in Schistosoma mansoni. PLoS Negl. Trop. Dis., 2011, 5, e1425.
74. Kumar, S.; Molina-Cruz, A.; Gupta, L.; Rodrigues, J.; Barillas-Mury, C. A peroxidase/dual oxidase system modulates midgut epithelial immunity in Anopheles gambiae. Science, 2010, 327, 1644-1648.
75. Grab, D.J.; Garcia-Garcia, J.C.; Nikolskaia, O.V.; Kim, Y.V.; Brown, A.; Pardo, C.A.; Zhang, Y.; Becker, K.G.; Wilson, B.A. de A.; Lima, A.P.; Scharfstein, J.; Dumler. J.S. Protease activated receptor signaling is required for African trypanosome traversal of human brain microvascular endothelial cells. PLoS Negl. Trop. Dis., 2009, 3, e479.
76. Maxwell, M.M.; Pasinelli, P.; Kazantsev, A.G.; Brown, R.H. Jr. RNA interference-mediated silencing of mutant superoxide dismutase rescues cyclosporin A-induced death in cultured neuroblastoma cells. Proc. Natl. Acad. Sci. U.S. A, 2004, 101, 3178-3183.
77. Raoul, C.; Abbas-Terki, T.; Bensadoun, J.C.; Guillot, S.; Haase, G.; Szulc, J.; Henderson, C.E.; Aebischer, P. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat. Med., 2005, 11, 423-428.
78. Ralph, G.S.; Radcliffe, P.A.; Day, D.M.; Carthy, J.M.; Leroux, M.A.; Lee, D.C.; Wong, L.F.; Bilsland, L.G.; Greensmith, L.; Kingsman, S.M.; Mitrophanous, K.A.; Mazarakis, N.D.; Azzouz, M. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat. Med., 2005, 11, 429-433.
79. Saito, Y.; Yokota, T.; Mitani, T.; Ito, K.; Anzai, M.; Miyagishi, M.; Taira, K.; Mizusawa, H. Transgenic small interfering RNA halts amyotrophic lateral sclerosis in a mouse model. J. Biol. Chem., 2005, 280, 42826-42830.
80. Xia. X.; Zhou, H.; Huang, Y.; Xu, Z. Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo. Neurobiol. Dis., 2006, 23, 578-586.
81. Locatelli, F.; Corti, S.; Papadimitriou, D.; Fortunato, F.; Del Bo, R.; Donadoni, C.; Nizzardo, M.; Nardini, M.; Salani, S.; Ghezzi, S.; Strazzer, S.; Bresolin, N.; Comi, G.P. Fas small interfering RNA reduces motoneuron death in amyotrophic lateral sclerosis mice. Ann. Neurol., 2007, 62, 81-92.
82. Xie, J.; Awad, K.S.; Guo, Q. RNAi knockdown of Par-4 inhibits neurosynaptic degeneration in ALS-linked mice. J. Neurochem., 2005, 92, 59-71.
83. Goehler, H.; Lalowski. M.; Stelzl, U.; Waelter, S.; Stroedicke, M.; Worm, U.; Droege, A.; Lindenberg, K.S.; Knoblich, M.; Haenig, C.; Herbst, M.; Suopanki, J.; Scherzinger, E.; Abraham, C.; Bauer, B.; Hasenbank, R.; Fritzsche, A.; Ludewig, A.H.; Büssow, K.; Coleman, S.H.; Gutekunst, C.A.; Landwehrmeyer, B.G.; Lehrach, H.; Wanker, E.E. A protein interaction network links GIT1, an enhancer of huntingtin aggregation, to Huntington's disease. Mol. Cell., 2004, 15, 853-865.
84. Qiu, Z.; Norflus, F.; Singh, B.; Swindell, M.K.; Buzescu, R.; Bejarano, M.; Chopra, R.; Zucker, B.; Benn, C.L.; DiRocco, D.P.; Cha, J.H.; Ferrante, R.J.; Hersch, S.M. Sp1 is up-regulated in cellular and transgenic models of Huntington disease, and its reduction is neuroprotective. J. Biol. Chem., 2006, 281, 16672-16680.
85. Harper, S.Q.; Staber, P.D.; He, X.; Eliason, S.L.; Martins, I.H.; Mao, Q.; Yang, L.; Kotin, R.M.; Paulson, H.L.; Davidson, B.L. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc. Natl. Acad. Sci. U.S.A., 2005, 102, 5820.
86. Wang, Y.L.; Liu, W.; Wada, E.; Murata, M.; Wada, K.; Kanazawa, I. Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA. Neurosci. Res., 2005, 53, 241-249.
87. Rodriguez-Lebron, E.; Denovan-Wright, E.M.; Nash, K.; Lewin, A.S.; Mandel, R.J. Intrastriatal rAAV-mediated delivery of antihuntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol. Ther., 2005, 12, 618-633.
88. Machida, Y.; Okada, T.; Kurosawa, M.; Oyama, F.; Ozawa, K.; Nukina, N. rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse. Biochem. Biophys. Res. Commun., 2006, 343, 190-197.
89. Huang, B.; Schiefer, J.; Sass, C.; Landwehrmeyer, G.B.; Kosinski, C.M.; Kochanek, S. High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo. Hum. Gene Ther., 2007, 18, 303-311.
90. DiFiglia, M.; Sena-Esteves, M.; Chase, K.; Sapp, E.; Pfister, E.; Sass, M.; Yoder, J.; Reeves, P.; Pandey, R.K.; Rajeev, K.G.; Manoharan, M.; Sah, D.W.; Zamore, P.D.; Aronin, N. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits. Proc. Natl. Acad. Sci. U.S.A., 2007, 104, 17204-17209.
91. Horvath, L.; van Marion, I.; Taï, K.; Nielsen, T.T.; Lundberg, C.J. Knockdown of GAD67 protein levels normalizes neuronal activity in a rat model of Parkinson's disease. Gene Med., 2011, 13, 188-197.
92. de Yñigo-Mojado, L.; Martín-Ruíz, I.; Sutherland, J.D. Efficient allele-specific targeting of LRRK2 R1441 mutations mediated by RNAi. PLoS One., 2011, 6, e21352.
93. Gerard, M.; Deleersnijder, A.; Daniëls, V.; Schreurs, S.; Munck, S.; Reumers, V.; Pottel, H.; Engelborghs, Y.; Van den Haute, C.; Taymans, J.M.; Debyser. Z.; Baekelandt. V. Inhibition of FK506 binding proteins reduces alpha-synuclein aggregation and Parkinson's disease-like pathology. J. Neurosci., 2010, 30, 2454-2463.
94. He, G.; Luo, W.; Li, P.; Remmers, C.; Netzer, W.J.; Hendrick, J.; Bettayeb, K.; Flajolet, M.; Gorelick, F.; Wennogle, L.P.; Greengard, P. Gamma-secretase activating protein is a therapeutic target for Alzheimer's disease. Nature, 2010, 467, 95-98.
95. López-Tobón, A.; Castro-Álvarez. J.F.; Piedrahita, D.; Boudreau, R.L.; Gallego-Gómez, J.C.; Cardona-Gómez, G.P. Silencing of CDK5 as potential therapy for Alzheimer's disease. Rev. Neurosci., 2011, 22, 143-152.
96. Hu, J.; Gagnon, K.T.; Liu. J.; Watts. J.K.; Syeda-Nawaz, J.; Bennett, C.F.; Swayze, E.E.; Randolph, J.; Chattopadhyaya, J.; Corey, D.R. Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs. Biol. Chem., 2011, 392, 315-325.
97. Alves, S.; Nascimento-Ferreira, I.; Dufour, N.; Hassig, R.; Auregan, G.; Nóbrega, C.; Brouillet, E.; Hantraye, P.; Pedroso de Lima, M.C.; Déglon, N.; de Almeida, L.P. Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3? Hum. Mol. Genet., 2010, 19, 2380-2394.
98. Nakano, I.; Masterman-Smith, M.; Saigusa, K.; Paucar, A.A.; Horvath, S.; Shoemaker, L.; Watanabe, M.; Negro, A.; Bajpai, R.; Howes, A.; Lelievre, V.; Waschek, J.A.; Lazareff, J.A.; Freije, W.A.; Liau, L.M.; Gilbertson, R.J.; Cloughesy, T.F.; Geschwind, D.H.; Nelson, S.F.; Mischel, P.S.; Terskikh, A.V.; Kornblum, H.I. Maternal embryonic leucine zipper kinase is a key regulator of the proliferation of malignant brain tumors, including brain tumor stem cells. J. Neurosci. Res., 2008, 86, 48.
99. Horvath, S.; Zhang, B.; Carlson, M.; Lu, K.V.; Zhu, S.; Felciano, R.M.; Laurance, M.F.; Zhao, W.; Qi, S.; Chen, Z.; Lee, Y.; Scheck, A.C.; Liau, L.M.; Wu, H.; Geschwind, D.H.; Febbo, P.G.; Kornblum, H.I.; Cloughesy, T.F.; Nelson, S.F.; Mischel, P.S. Analysis of oncogenic signaling networks in glioblastoma identifies ASPM as a molecular target. Proc. Natl. Acad. Sci. U.S.A., 2006, 103, 17402-17407.
100. Patry, C.; Bouchard, L.; Labrecque, P.; Gendron, D.; Lemieux, B.; Toutant, J.; Lapointe, E.; Wellinger, R.; Chabot, B. Small interfering RNA-mediated reduction in heterogeneous nuclear ribonucleoparticule A1/A2 proteins induces apoptosis in human cancer cells but not in normal mortal cell lines. Cancer Res., 2003, 63, 7679-7688.
101. Folkman, J. Tumor angiogenesis: therapeutic implications. N. Engl. J. Med., 1971, 285, 1182-1186.
102. Sun, L.; Hui, A.M.; Su, Q.; Vortmeyer, A.; Kotliarov, Y.; Pastorino, S.; Passaniti, A.; Menon, J.; Walling, J.; Bailey, R.; Rosenblum, M.; Mikkelsen, T.; Fine, H.A. Neuronal and gliomaderived stem cell factor induces angiogenesis within the brain. Cancer Cell, 2006, 9, 287-300.
103. Peters, K.G.; Coogan, A.; Berry, D.; Marks, J.; Iglehart, J.D.; Kontos, C.D.; Rao, P.; Sankar, S.; Trogan, E. Expression of Tie2/Tek in breast tumour vasculature provides a new marker for evaluation of tumour angiogenesis. Br. J. Cancer, 1998, 77, 51-56.
104. Gondi, C.S.; Lakka, S.S.; Dinh, D.H.; Olivero, W.C.; Gujrati, M.; Rao, J.S. Intraperitoneal injection of a hairpin RNA-expressing plasmid targeting urokinase-type plasminogen activator (uPA) receptor and uPA retards angiogenesis and inhibits intracranial tumor growth in nude mice. Clin. Cancer Res., 2007, 13, 4051-4060.
105. Pyrko, P.; Schönthal, A.H.; Hofman, F.M.; Chen, T.C.; Lee, A.S. The unfolded protein response regulator GRP78/BiP as a novel target for increasing chemosensitivity in malignant gliomas. Cancer Res., 2007, 67, 9809-9816.
106. Goidts, V.; Bageritz, J.; Puccio, L.; Nakata, S.; Zapatka, M.; Barbus, S.; Toedt, G.; Campos, B.; Korshunov, A.; Momma, S.; Van Schaftingen, E.; Reifenberger, G.; Herold-Mende, C.; Lichter, P.; Radlwimmer, B. RNAi screening in glioma stem-like cells identifies PFKFB4 as a key molecule important for cancer cell survival. Oncogene, 2012, 31(27). 3235-43.
107. Wurdak, H.; Zhu, S.; Romero, A.; Lorger, M.; Watson, J.; Chiang, C.Y.; Zhang, J.; Natu, V.S.; Lairson, L.L.; Walker, J.R.; Trussell, C.M.; Harsh, G.R.; Vogel, H.; Felding-Habermann, B.; Orth, A.P.; Miraglia, L.J.; Rines. D.R.; Skirboll, S.L.; Schultz, P.G. An RNAi screen identifies TRRAP as a regulator of brain tumor-initiating cell differentiation. Cell Stem Cell, 2010, 6, 37-47.
108. Sheng, Z.; Li, L,; Zhu, L.J.; Smith, T.W.; Demers, A.; Ross, A.H.; Moser, R.P.; Green, M.R. A genome-wide RNA interference screen reveals an essential CREB3L2-ATF5-MCL1 survival pathway in malignant glioma with therapeutic implications. Nat Med., 2010, 16, 671-677.
109. Kim, Y.W.; Liu, T.J.; Koul, D.; Tiao, N.; Feroze, A.H.; Wang, J.; Powis, G.; Yung, W.K. Identification of novel synergistic targets for rational drug combinations with PI3 kinase inhibitors using siRNA synthetic lethality screening against GBM. Neuro Oncol., 2011, 13, 367-375.
110. Davis, M.E.; Zuckerman, J.E.; Choi, C.H.; Seligson, D.; Tolcher, A.; Alabi, C.A.; Yen, Y.; Heidel, J.D.; Ribas, A. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature, 2010, 464, 1067-1070.
111. Musatov, S.; Chen, W.; Pfaff, D.W.; Kaplitt, M.G.; Ogawa, S. RNAi-mediated silencing of estrogen receptor {alpha} in the ventromedial nucleus of hypothalamus abolishes female sexual behaviors. Proc. Natl. Acad. Sci. U.S.A., 2006, 103, 10456-10460.
112. Lasek, A.W.; Janak, P.H.; He, L.; Whistler, J.L.; Heberlein, U. Downregulation of mu opioid receptor by RNA interference in the ventral tegmental area reduces ethanol consumption in mice. Genes Brain Behav., 2007, 6, 728-735.
113. Bahi, A.; Boyer, F.; Bussard, G.; Dreyer, J.L. Silencing dopamine D3-receptors in the nucleus accumbens shell in vivo induces changes in cocaine-induced hyperlocomotion. Eur. J. Neurosci., 2005, 21, 3415-3426.
114. Thakker, D.R.; Natt, F.; Hüsken, D.; van der Putten, H.; Maier, R.; Hoyer, D.; Cryan, J.F. siRNA-mediated knockdown of the serotonin transporter in the adult mouse brain. Mol. Psychiatry, 2005, 10, 782-789.
115. Christoph, T.; Grünweller, A.; Mika, J.; Schäfer, M.K.; Wade, E.J.; Weihe, E.; Erdmann, V.A.; Frank, R.; Gillen, C.; Kurreck, J. Silencing of vanilloid receptor TRPV1 by RNAi reduces neuropathic and visceral pain in vivo. Biochem. Biophys. Res. Commun., 2006, 350, 238-243.
116. Kasama, S.; Kawakubo, M.; Suzuki, T.; Nishizawa, T.; Ishida, A.; Nakayama, J. RNA interference-mediated knock-down of transient receptor potential vanilloid 1 prevents forepaw inflammatory hyperalgesia in rat. Eur. J. Neurosci., 2007, 25, 2956-2963.
117. Altier, C.; Dale, C.S.; Kisilevsky, A.E.; Chapman, K.; Castiglioni, A.J.; Matthews, E.A.; Evans, R.M.; Dickenson, A.H.; Lipscombe, D.; Vergnolle, N.; Zamponi, G.W. Differential role of N-type calcium channel splice isoforms in pain. J. Neurosci, 2007, 27, 6363-63673.
118. Yang. C.; Gagnon, D.; Vachon, P.; Tremblay, A.; Levy, E.; Massie, B.; Michaud, J.L. Adenoviral-mediated modulation of Sim1 expression in the paraventricular nucleus affects food intake. J. Neurosci., 2006, 26, 7116-7120.
119. Shishkina, G.T.; Kalinina, T.S.; Dygalo, N.N. Attenuation of μ2Aadrenergic receptor expression in neonatal rat brain by RNA interference or antisense oligonucleotide reduced anxiety in adulthood. Neuroscience, 2004, 129, 521-528.
120. Hovatta, I.; Tennant, R.S.; Helton, R.; Marr, R.A.; Singer, O.; Redwine, J.M.; Ellison, J.A.; Schadt, E.E.; Verma, I.M.; Lockhart, D.J.; Barlow, C. Glyoxalase 1 and glutathione reductase 1 regulate anxiety in mice. Nature, 2005, 438, 662-666.
121. Johnson, P.L.; Truitt, W.; Fitz, S.D.; Minick, P.E.; Dietrich, A.; Sanghani, S.; Träskman-Bendz, L.; Goddard, A.W.; Brundin, L.; Shekhar, A.; A key role for orexin in panic anxiety. Nat. Med., 2010, 16, 111-115.
122. Kim, K.S.; Lee, K.W.; Baek, I.S.; Lim, C.M.; Krishnan. V.; Lee, J.K.; Nestler, E.J.; Han, P.L. Adenylyl cyclase-5 activity in the nucleus accumbens regulates anxiety-related behavior. J. Neurochem., 2008, 107, 105-115.
123. Bortolozzi, A.; Castañé, A.; Semakova, J.; Santana, N.; Alvarado, G.; Cortés, R.; Ferrés-Coy, A.; Fernández, G.; Carmona, M.C.; Toth, M.; Perales, J.C.; Montefeltro, A.; Artigas, F. Selective siRNA-mediated suppression of 5-HT(1A) autoreceptors evokes strong anti-depressant-like effects. Mol. Psychiatry, 2012, 17(6), 612-23.
124. Bartel, D.P. MicroRNAs: genomics, biogenesis, mechanism, and function. Cell, 2004, 116, 281-297.
125. Sontheimer, E.J.; Carthew, R.W. Silence from within: endogenous siRNAs and miRNAs. Cell, 2005, 122, 9-12.
126. Kiriakidou, M.; Tan, G.S.; Lamprinaki, S.; De Planell-Saguer, M.; Nelson, P.T.; Mourelatos, Z. An mRNA m7G cap binding-like motif within human Ago2 represses translation. Cell, 2007, 129, 1141-1151.
127. Mack, G.S. MicroRNA gets down to business. Nat. Biotechnol., 2007, 25, 631-638.
128. Niu, Q.W.; Lin, S.S.; Reyes, J.L.; Chen, K.C.; Wu, H.W.; Yeh, S.D.; Chua, N.H. Nat. Biotechnol., 2006, 24, 1420.
129. Calin, G.A.; Dumitru, C.D.; Shimizu, M.; Bichi, R.; Zupo, S.; Noch, E.; Aldler, H.; Rattan, S.; Keating, M.; Rai, K.; Rassenti, L.; Kipps, T.; Negrini, M.; Bullrich, F.; Croce, C.M. Frequent deletions and down-regulation of micro-RNA genes miR15 and miR16 at 13q14 in chronic lymphocytic leukemia. Proc. Natl. Acad. Sci. U.S.A., 2002, 99, 15524-15529.
130. Shi, B.; Sepp-Lorenzino, L.; Prisco, M.; Linsley, P.; deAngelis, T.; Baserga, R. Micro RNA 145 targets the insulin receptor substrate-1 and inhibits the growth of colon cancer cells. J. Biol. Chem., 2007, 282, 32582-32590.
131. Musiyenko, A.; Bitko, V.; Barik, S. Ectopic expression of miR-126*, an intronic product of the vascular endothelial EGF-like 7 gene, regulates prostein translation and invasiveness of prostate cancer LNCaP cells. J. Mol. Med., 2008, 86, 313-322.
132. Tsuda, N.; Ishiyama, S.; Li, Y.; Ioannides, C.G.; Abbruzzese, J.L.; Chang, D.Z. Synthetic microRNA designed to target glioma associated antigen 1 transcription factor inhibits division and induces late apoptosis in pancreatic tumor cells. Clin. Cancer Res., 2006, 12, 6557-6564.
133. Nan, Y.; Han, L.; Zhang, A.; Wang, G.; Jia, Z.; Yang, Y.; Yue, X.; Pu, P.; Zhong, Y.; Kang, C. MiRNA-451 plays a role as tumor suppressor in human glioma cells. Brain Res., 2010, 1359, 14-21.
134. Schratt, G.M.; Tuebing, F.; Nigh, E.A.; Kane, C.G.; Sabatini, M.E.; Kiebler, M.; Greenberg, M.E. A brain-specific microRNA regulates dendritic spine development. Nature, 2006, 439, 283-289.
135. Stern-Ginossar, N.; Elefant, N.; Zimmermann, A.; Wolf, D.G.; Saleh, N.; Biton, M.; Horwitz, E.; Prokocimer, Z.; Prichard, M.; Hahn, G.; Goldman-Wohl, D.; Greenfield, C.; Yagel, S.; Hengel, H.; Altuvia, Y.; Margalit, H.; Mandelboim, O. Host immune system gene targeting by a viral miRNA. Science, 2007, 317, 376-381.
136. Lecellier, C.H.; Dunoyer, P.; Arar, K.; Lehmann-Che, J.; Eyquem, S.; Himber, C.; Saïb, A.; Voinnet, O. A cellular microRNA mediates antiviral defense in human cells. Science, 2005, 308, 557-560.
137. Sullivan, C.S.; Grundhoff, A.T.; Tevethia, S.; Pipas, J.M.;, Ganem, D. SV40-encoded microRNAs regulate viral gene expression and reduce susceptibility to cytotoxic T cells. Nature, 2005, 435, 682-686.
138. Ebert, M.S.; Neilson, J.R.; Sharp, P.A. MicroRNA sponges: competitive inhibitors of small RNAs in mammalian cells. Nat. Methods, 2007, 4, 721-726.
139. Ebert, M.S.; Sharp, P.A. MicroRNA sponges: progress and possibilities. RNA, 2010, 16, 2043-2050.
140. Kluiver, J.; Gibcus, J.H.; Hettinga, C.; Adema, A.; Richter, M.K.; Halsema, N.; Slezak-Prochazka, I.; Ding, Y.; Kroesen, B.J.; van den Berg, A. Rapid generation of microRNA sponges for microRNA inhibition. PLoS One, 2012, 7, e29275.
141. Li, L.C.; Okino, S.T.; Zhao, H.; Pookot, D.; Place, R.F.; Urakami, S.; Enokida, H.; Dahiya, R. Small dsRNAs induce transcriptional activation in human cells. Proc. Natl. Acad. Sci. U.S.A., 2006, 103, 17337-17342.
142. Janowski, B.A.; Younger, S.T.; Hardy, D.B.; Ram, R.; Huffman, K.E.; Corey, D.R. Activating gene expression in mammalian cells with promoter-targeted duplex RNAs. Nat. Chem. Biol., 2007, 3, 166-173.
143. Check, E. RNA interference: hitting the on switch. Nature, 2007, 448, 855-858.
144. Huang, V.; Qin, Y.; Wang, J.; Wang, X.; Place, R.F.; Lin, G.; Lue, T.F.; Li, L.C. RNAa is conserved in mammalian cells. PLoS One, 2010, 5, e8848.
145. Place, R.F.; Li, L.C.; Pookot, D.; Noonan, E.J.; Dahiya, R. MicroRNA-373 induces expression of genes with complementary promoter sequences. Proc. Natl. Acad. Sci. U.S.A., 2008, 105, 1608-1613.
146. Noma, K.; Sugiyama, T.; Cam, H.; Verdel, A.; Zofall, M.; Jia, S.; Moazed, D.; Grewal, S.I. RITS acts in cis to promote RNA interference-mediated transcriptional and post-transcriptional silencing. Nat. Genet., 2004, 36, 1174-1180.
147. Morris, K.V.; Chan, S.W.; Jacobsen, S.E.; Looney, D.J. Small interfering RNA-induced transcriptional gene silencing in human cells. Science, 2004, 305, 1289-1292.
148. Vastenhouw, N.L.; Brunschwig, K.; Okihara, K.L.; Müller, F.; Tijsterman, M.; Plasterk, R.H. Gene expression: long-term gene silencing by RNAi. Nature, 2006, 24, 442, 882.
149. Zhang, H.Y.; Du, Q.; Wahlestedt, C.; Liang, Z. RNA Interference with chemically modified siRNA. Curr. Top. Med. Chem., 2006, 6, 893-900.
150. Hoerter, JA.; Walter, N.G. Chemical modification resolves the asymmetry of siRNA strand degradation in human blood serum. RNA, 2007, 13, 1887-1893.
151. Sipa, K.; Sochacka, E.; Kazmierczak-Baranska, J.; Maszewska, M.; Janicka, M.; Nowak, G.; Nawrot, B. Effect of base modifications on structure, thermodynamic stability, and gene silencing activity of short interfering RNA. RNA, 2007, 13, 1301-1316.
152. Birmingham, A.; Anderson, E.M.; Reynolds, A.; Ilsley-Tyree, D.; Leake, D.; Fedorov, Y.; Baskerville, S.; Maksimova, E.; Robinson, K.; Karpilow, J.; Marshall, W.S.; Khvorova, A. 3' UTR seed matches, but not overall identity, are associated with RNAi offtargets. Nat. Methods, 2006, 3, 199-204.
153. Lewis, B.P.; Burge, C.B.; Bartel, D.P. Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets. Cell, 2005, 120, 15-20.
154. Grimson, A.; Farh, K.K.; Johnston, W.K.; Garrett-Engele, P.; Lim, L.P.; Bartel, D.P. MicroRNA targeting specificity in mammals: determinants beyond seed pairing. Mol. Cell, 2007, 27, 91-105.
155. Towne, C.; Setola, V.; Schneider, B.L.; Aebischer, P. Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic benefit in fALS mice. Mol. Ther., 2011, 19, 274-283.
156. Check, E. RNA therapy beckons as firms prepare for clinical trials. Nature, 2004, 429, 792.
157. Check, E. Firm sets sights on gene silencing to protect vision. Nature, 2004, 430, 819.
158. Singer, O.; Marr, RA.; Rockenstein, E.; Crews, L.; Coufal, NG.; Gage, FH.; Verma, I.M.; Masliah, E. Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model. Nat. Neurosci., 2005, 8, 1343-1349.
159. Xie, Z.; Romano, D.M.; Tanzi, R.E. RNA interference-mediated silencing of X11alpha and X11beta attenuates amyloid beta-protein levels via differential effects on beta-amyloid precursor protein processing. J. Biol. Chem., 2005, 280, 15413-15421.
160. Hayashita-Kinoh, H.; Yamada, M.; Yokota, T.; Mizuno, Y.; Mochizuki, H. Down-regulation of alpha-synuclein expression can rescue dopaminergic cells from cell death in the substantia nigra of Parkinson's disease rat model. Biochem. Biophys. Res. Commun., 2006, 341, 1088-1095.
161. Sapru M.K.; Yates J.W.; Hogan S.; Jiang L.; Halter J.; Bohn M.C. Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi. Exp. Neurol., 2006, 198, 382-390.
162. Outeiro, T.F.; Kontopoulos, E.; Altmann, S.M.; Kufareva, I.; Strathearn, K.E.; Amore, A.M.; Volk, C.B.; Maxwell, M.M.; Rochet, J.C.; McLean, P.J.; Young, A.B.; Abagyan, R.; Feany, M.B.; Hyman, B.T.; Kazantsev, A.G. Sirtuin 2 inhibitors rescue alpha-synuclein-mediated toxicity in models of Parkinson's disease. Science, 2007, 317, 516-519.
163. Xia, H.; Mao, Q.; Eliason, S.L.; Harper, S.Q.; Martins, I.H.; Orr, H.T.; Paulson, H.L.; Yang, L.; Kotin, R.M.; Davidson, B.L. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat. Med., 2004, 10, 816-820.
164. Murakami, M.; Ota, T.; Nukuzuma, S.; Takegami, T. Inhibitory effect of RNAi on Japanese encephalitis virus replication in vitro and in vivo. Microbiol. Immunol., 2005, 49, 1047-1056.
165. Muylaert, I.; Elias, P. Knockdown of DNA ligase IV/XRCC4 by RNA interference inhibits herpes simplex virus type I DNA replication. J. Biol. Chem., 2007, 282, 10865-10872.
166. Bai, F.; Wang, T.; Pal, U.; Bao, F.; Gould, L.H.; Fikrig, E. Use of RNA interference to prevent lethal murine west nile virus infection. J. Infect. Dis., 2005, 191, 1148-1154.