Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism

Chemistry and Biology

Volumn 17, Issue 11, 2010, Pages 1183-1188

  Hu, Jiaxin ^a   Liu, Jing ^a   Corey, David R ^a  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

HD PROTEIN, HUMAN; MICRORNA; NERVE PROTEIN; NUCLEAR PROTEIN; SMALL INTERFERING RNA;

ALLELE; ARTICLE; BASE MISPAIRING; CELL LINE; DRUG ANTAGONISM; GENETICS; HUMAN; HUNTINGTON CHOREA; METABOLISM; MUTATION; NUCLEOTIDE SEQUENCE; RNA INTERFERENCE;

ALLELES; BASE PAIR MISMATCH; BASE SEQUENCE; CELL LINE; HUMANS; HUNTINGTON DISEASE; MICRORNAS; MUTATION; NERVE TISSUE PROTEINS; NUCLEAR PROTEINS; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 78649359013     PISSN: 10745521     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.chembiol.2010.10.013     Document Type: Article

References (36)

1. R.L. Boudreau, J.L. McBride, I. Martins, S. Shen, Y. Xing, B.J. Carter, and B.L. Davidson Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice Mol. Ther. 17 2009 1053 1063
2. E.B. De Souza, S.T. Cload, P.S. Pendergrast, and D.W. Sah Novel therapeutic modalities to address nondrugable protein interaction targets Neuropsychopharmacology 34 2009 142 158
3. M. DiFiglia, M. Sena-Esteves, K. Chase, E. Sapp, E. Pfister, M. Sass, J. Yoder, P. Reeves, R.K. Pandey, and K.G. Rajeev Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits Proc. Natl. Acad. Sci. USA 104 2007 17204 17209
4. V. Drouet, V. Perrin, R. Hassig, N. Dufour, G. Auregan, S. Alves, G. Bonvento, E. Brouillet, R. Luthi-Carter, P. Hantraye, and N. Deglon Sustained effects of nonallele-specific Huntingtin silencing Ann. Neurol. 65 2009 276 285
5. Q. Du, H. Thonberg, J. Wang, C. Wahlestedt, and Z. Liang A systematic analysis of the silencing effects of an active siRNA at all single-nucleotide mismatched target sites Nucleic Acids Res. 33 2005 1671 1677
6. M. Duyao, C. Ambrose, R. Myers, A. Novelletto, F. Persichetti, M. Frontali, S. Folstein, C. Ross, M. Franz, and M. Abbott Trinucleotide repeat length instability and age of onset in Huntington's disease Nat. Genet. 4 1993 387 392 (Pubitemid 23231488)
7. W. Filipowicz, S.N. Bhattacharyya, and N. Sonenberg Mechanisms of post-transcriptional regulation by microRNAs: are the answers in sight? Nat. Rev. Genet. 9 2008 102 114
8. K.T. Gagnon HD therapeutics - CHDI fifth annual conference IDrugs 13 2010 219 233
9. K.T. Gagnon, H.M. Pendergraff, G.F. Deleavey, E.E. Swayze, P. Potier, J. Randolph, E.B. Roesch, J. Chattopadhyaya, M.J. Damha, and C.F. Bennet Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat Biochemistry 2010 in press. Published online October 28, 2010.
10. A. Grimson, K.K. Farh, W.K. Johnston, P. Garrett-Engele, L.P. Lim, and D.P. Bartel MicroRNA targeting specificity in mammals: determinants beyond seed pairing Mol. Cell 27 2007 91 105
11. S.Q. Harper, P.D. Staber, X. He, S.L. Eliason, I.H. Martins, Q. Mao, L. Yang, R.M. Kotin, H.L. Paulson, and B.L. Davidson RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model Proc. Natl. Acad. Sci. USA 102 2005 5820 5825
12. J. Hu, M. Matsui, and D.R. Corey Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA Ann. N. Y. Acad. Sci. 1175 2009 24 31
13. J. Hu, D.W. Dodd, R.H. Hudson, and D.R. Corey Cellular localization and allele-selective inhibition of mutant huntingtin protein by peptide nucleic acid oligomers containing the fluorescent nucleobase [bis-o-(aminoethoxy)phenyl] pyrrolocytosine Bioorg. Med. Chem. Lett. 19 2009 6181 6184
14. J. Hu, M. Matsui, K.T. Gagnon, J.C. Schwartz, S. Gabillet, K. Arar, J. Wu, I. Bezprozvanny, and D.R. Corey Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs Nat. Biotechnol. 27 2009 478 484
15. H. Knudsen, and P.E. Nielsen Antisense properties of duplex- and triplex-forming PNAs Nucleic Acids Res. 24 1996 494 500
16. T. Koch, C. Rosenbohm, H.F. Hanson, B. Hansen, E.M. Straarup, and S. Kauppinen Locked nucleic acid: properties and therapeutic aspects J. Kurreck, Therapeutic Oligonucleotides 2008 RSC Biomolecular Sciences, Royal Society of Chemistry Cambridge, UK 103 141
17. B. Kremer, P. Goldberg, S.E. Andrew, J. Theilmann, H. Telenius, J. Zeisler, F. Squitieri, B. Lin, A. Bassett, and E. Almqvist A worldwide study of the Huntington's disease mutation. The sensitivity and specificity of measuring CAG repeats N. Engl. J. Med. 330 1994 1401 1406
18. J. Kurreck RNA interference: from basic research to therapeutic applications Angew. Chem. Int. Ed. Engl. 48 2009 1378 1398
19. J. Liu, M.A. Carmell, F.V. Rivas, C.G. Marsden, J.M. Thomson, J.J. Song, S.M. Hammond, L. Joshua-Tor, and G.J. Hannon Argonaute2 is the catalytic engine of mammalian RNAi Science 305 2004 1437 1441
20. M.S. Lombardi, L. Jaspers, C. Spronkmans, C. Gellera, F. Taroni, E. Di Maria, S.D. Donato, and W.F. Kaemmerer A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference Exp. Neurol. 217 2009 312 319
21. M.E. MacDonald, C.M. Ambrose, M.P. Duyao, R.H. Myers, C. Lin, L. Srinidhi, G. Barnes, S.A. Taylor, M. James, and N. Groot A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes Cell 72 1993 971 983
22. V.M. Miller, H. Xia, G.L. Marrs, C.M. Gouvion, G. Lee, B.L. Davidson, and H.L. Paulson Allele-specific silencing of dominant disease genes Proc. Natl. Acad. Sci. USA 100 2003 7195 7200
23. J. Nasir, S.B. Floresco, J.R. O'Kusky, V.M. Diewert, J.M. Richman, J. Zeisler, A. Borowski, J.D. Marth, A.G. Phillips, and M.R. Hayden Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes Cell 81 1995 811 823
24. K. Omi, N.S. Hachiya, K. Tokunaga, and K. Kaneko siRNA-mediated inhibition of endogenous Huntington disease gene expression induces an aberrant configuration of the ER network in vitro Biochem. Biophys. Res. Commun. 338 2005 1229 1235
25. E.L. Pfister, and P.D. Zamore Huntington's disease: silencing a brutal killer Exp. Neurol. 220 2009 226 229
26. E.L. Pfister, L. Kennington, J. Straubhaar, S. Wagh, W. Liu, M. DiFiglia, B. Landwehrmeyer, J.P. Vonsattel, P.D. Zamore, and N. Aronin Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients Curr. Biol. 19 2009 774 778
27. P.H. Reddy, M. Williams, V. Charles, L. Garrett, L. Pike-Buchanan, W.O. Whetsell Jr., G. Miller, and D.A. Tagle Behavioural abnormalities and selective neuronal loss in HD transgenic mice expressing mutated full-length HD cDNA Nat. Genet. 20 1998 198 202
28. I. Sanchez, C. Mahlke, and J. Yuan Pivotal role of oligomerization in expanded polyglutamine neurodegenerative disorders Nature 421 2003 373 379
29. D.S. Schwarz, H. Ding, L. Kennington, J.T. Moore, J. Schelter, J. Burchard, P.S. Linsley, N. Aronin, Z. Xu, and P.D. Zamore Designing siRNA that distinguish between genes that differ by a single nucleotide PLoS Genet. 2 2006 e140
30. R.A. Smith, T.M. Miller, K. Yamanaka, B.P. Monia, T.P. Condon, G. Hung, C.S. Lobsiger, C.M. Ward, M. McAlonis-Downes, and H. Wei Antisense oligonucleotide therapy for neurodegenerative disease J. Clin. Invest. 116 2006 2290 2296
31. P.H. van Bilsen, L. Jaspers, M.S. Lombardi, J.C. Odekerken, E.N. Burright, and W.F. Kaemmerer Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts Hum. Gene Ther. 19 2008 710 719
32. F.O. Walker Huntington's disease Lancet 369 2007 218 228
33. Y. Wang, S. Juranek, H. Li, G. Sheng, T. Tuschl, and D.J. Patel Structure of an argonaute silencing complex with a seed-containing guide DNA and target RNA duplex Nature 456 2008 921 926
34. T.M. Wheeler, K. Sobczak, J.D. Lueck, R.J. Osborne, X. Lin, R.T. Dirksen, and C.A. Thornton Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA Science 325 2009 336 339
35. J.K. White, W. Auerbach, M.P. Duyao, J.P. Vonsattel, J.F. Gusella, A.L. Joyner, and M.E. MacDonald Huntingtin is required for neurogenesis and is not impaired by the Huntington's disease CAG expansion Nat. Genet. 17 1997 404 410
36. Y. Zhang, J. Engelman, and R.M. Friedlander Allele-specific silencing of mutant Huntington's disease gene J. Neurochem. 108 2009 82 90