Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy

Nucleic Acids Research

Volumn 43, Issue 6, 2015, Pages 3318-3331

  Wojtkowiak Szlachcic, Agnieszka ^a   Taylor, Katarzyna ^a   Stepniak Konieczna, Ewa ^a   Sznajder, Lukasz J ^a   Mykowska, Agnieszka ^a   Sroka, Joanna ^a   Thornton, Charles A ^b   Sobczak, Krzysztof ^a  

^a ADAM MICKIEWICZ UNIVERSITY   (Poland)

^b UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

LOCKED NUCLEIC ACID; MBNL1 PROTEIN; RNA BINDING PROTEIN; UNCLASSIFIED DRUG; 3' UNTRANSLATED REGION; ANTISENSE OLIGONUCLEOTIDE; DMPK PROTEIN, HUMAN; MBNL1 PROTEIN, HUMAN; MUTANT PROTEIN; MYOTONIC DYSTROPHY PROTEIN KINASE; OLIGONUCLEOTIDE;

ADULT; ALTERNATIVE RNA SPLICING; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BINDING AFFINITY; CELL PROLIFERATION; CONTROLLED STUDY; CUG REPEAT EXPANSION; DRUG BIOAVAILABILITY; DRUG DISTRIBUTION; DRUG EFFICACY; DRUG POTENCY; DRUG SCREENING; DRUG SPECIFICITY; EC50; GENE EXPRESSION; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MYOTONIC DYSTROPHY; NONHUMAN; PRIORITY JOURNAL; PROTEIN RNA BINDING; RNA DEGRADATION; RNA STRUCTURE; 3' UNTRANSLATED REGION; ANIMAL; CELL LINE; DISEASE MODEL; GENETICS; HEK293 CELL LINE; HELA CELL LINE; METABOLISM; NUCLEOTIDE SEQUENCE; TRANSGENIC MOUSE; TRINUCLEOTIDE REPEAT;

3' UNTRANSLATED REGIONS; ALTERNATIVE SPLICING; ANIMALS; BASE SEQUENCE; CELL LINE; DISEASE MODELS, ANIMAL; HEK293 CELLS; HELA CELLS; HUMANS; MICE; MICE, TRANSGENIC; MUTANT PROTEINS; MYOTONIC DYSTROPHY; MYOTONIN-PROTEIN KINASE; OLIGONUCLEOTIDES; OLIGONUCLEOTIDES, ANTISENSE; RNA-BINDING PROTEINS; TRINUCLEOTIDE REPEAT EXPANSION;

EID: 84941758187     PISSN: 03051048     EISSN: 13624962     Source Type: Journal    
DOI: 10.1093/nar/gkv163     Document Type: Article

References (112)

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70. Lee, J.E., Bennett, C.F. and Cooper, T.A. (2012) RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1. Proc. Natl. Acad. Sci. U.S.A., 109, 4221-4226.
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73. Wheeler, T.M., Sobczak, K., Lueck, J.D., Osborne, R.J., Lin, X., Dirksen, R.T. and Thornton, C.A. (2009) Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA. Science, 325, 336-339.
74. Konieczny, P., Stepniak-Konieczna, E. and Sobczak, K. (2014) MBNL proteins and their target RNAs, interaction and splicing regulation. Nucleic Acids Res., 42, 10873-10887.
75. Kaur, H., Babu, B.R. and Maiti, S. (2007) Perspectives on chemistry and therapeutic applications of Locked Nucleic Acid (LNA). Chem. Rev., 107, 4672-4697.
76. Cheng, C.J., Saltzman, W.M. and Slack, F.J. (2013) Canonical and non-canonical barriers facing antimiR cancer therapeutics. Curr. Med. Chem., 20, 3582-3593.
77. Jepsen, J.S., Sorensen, M.D. and Wengel, J. (2004) Locked nucleic acid: a potent nucleic acid analog in therapeutics and biotechnology. Oligonucleotides, 14, 130-146.
78. Rigo, F., Seth, P.P. and Bennett, C.F. (2014) Antisense oligonucleotide-based therapies for diseases caused by pre-mRNA processing defects. Adv. Exp. Med. Biol., 825, 303-352.
79. Burnett, J.C. and Rossi, J.J. (2012) RNA-based therapeutics: current progress and future prospects. Chem. Biol., 19, 60-71.
80. Fluiter, K., Mook, O.R. and Baas, F. (2009) The therapeutic potential of LNA-modified siRNAs: reduction of off-target effects by chemical modification of the siRNA sequence. Methods Mol. Biol., 487, 189-203.
81. Greenberger, L.M., Horak, I.D., Filpula, D., Sapra, P., Westergaard, M., Frydenlund, H.F., Albaek, C., Schroder, H. and Orum, H. (2008) A RNA antagonist of hypoxia-inducible factor-1alpha, EZN-2968, inhibits tumor cell growth. Mol. Cancer Ther., 7, 3598-3608.
82. Lanford, R.E., Hildebrandt-Eriksen, E.S., Petri, A., Persson, R., Lindow, M., Munk, M.E., Kauppinen, S. and Orum, H. (2010) Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science, 327, 198-201.
83. Hildebrandt-Eriksen, E.S, Aarup, V., Persson, R., Hansen, H.F., Munk, M.E. and Orum, H. (2012) A locked nucleic acid oligonucleotide targeting microRNA 122 is well-tolerated in cynomolgus monkeys. Nucleic Acid Ther., 22, 152-161.
84. Obad, S., dos Santos, C.O., Petri, A., Heidenblad, M., Broom, O., Ruse, C., Fu, C., Lindow, M., Stenvang, J., Straarup, E.M. et al. (2011) Silencing of microRNA families by seed-targeting tiny LNAs. Nat. Genet., 43, 371-378.
85. Hamshere, M.G., Newman, E.E., Alwazzan, M., Athwal, B.S. and Brook, J.D. (1997) Transcriptional abnormality in myotonic dystrophy affects DMPK but not neighboring genes. Proc. Natl. Acad. Sci. U. S. A., 94, 7394-7399.
86. Sabouri, L.A., Mahadevan, M.S., Narang, M., Lee, D.S., Surh, L.C. and Korneluk, R.G. (1993) Effect of the myotonic dystrophy (DM) mutation on mRNA levels of the DM gene. Nat. Genet., 4, 233-238.
87. de Mezer, M., Wojciechowska, M., Napierala, M., Sobczak, K. and Krzyzosiak, W.J. (2011) Mutant CAG repeats of Huntingtin transcript fold into hairpins, form nuclear foci and are targets for RNA interference. Nucleic Acids Res., 39, 3852-3863.
88. Wojciechowska, M., Taylor, K., Sobczak, K., Napierala, M. and Krzyzosiak, W.J. (2014) Small molecule kinase inhibitors alleviate different molecular features of myotonic dystrophy type 1. RNA Biol., 11. 742-754.
89. Mankodi, A., Logigian, E., Callahan, L., McClain, C., White, R., Henderson, D., Krym, M. and Thornton, C.A. (2000) Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat. Science, 289, 1769-1773.
90. Wheeler, T.M., Lueck, J.D., Swanson, M.S., Dirksen, R.T. and Thornton, C.A. (2007) Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy. J. Clin. Invest., 117, 3952-3957.
91. www.exiqon.com/ls/Pages/ExiqonTMPredictionTool.aspx.
92. Mykowska, A., Sobczak, K., Wojciechowska, M., Kozlowski, P. and Krzyzosiak, W.J. (2011) CAG repeats mimic CUG repeats in the misregulation of alternative splicing. Nucleic Acids Res., 39, 8938-8951.
93. Du, H., Cline, M.S., Osborne, R.J., Tuttle, D.L., Clark, T.A., Donohue, J.P., Hall, M.P., Shiue, L., Swanson, M.S., Thornton, C.A. et al. (2010) Aberrant alternative splicing and extracellular matrix gene expression in mouse models of myotonic dystrophy. Nat. Struct. Mol. Biol., 17, 187-193.
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