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Items: 1 to 20 of 138

1.

A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy.

Singh NN, Shishimorova M, Cao LC, Gangwani L, Singh RN.

RNA Biol. 2009 Jul-Aug;6(3):341-50. Epub 2009 Jul 14.

2.

Evolving concepts on human SMN pre-mRNA splicing.

Singh RN.

RNA Biol. 2007 Jan-Mar;4(1):7-10. Epub 2007 Jun 4.

PMID:
17592254
3.

A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes.

Singh NN, Seo J, Rahn SJ, Singh RN.

PLoS One. 2012;7(11):e49595. doi: 10.1371/journal.pone.0049595. Epub 2012 Nov 19.

4.
5.

Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions.

Singh NN, Lee BM, Singh RN.

Ann N Y Acad Sci. 2015 Apr;1341:176-87. doi: 10.1111/nyas.12727. Epub 2015 Feb 27.

6.

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

Hua Y, Vickers TA, Okunola HL, Bennett CF, Krainer AR.

Am J Hum Genet. 2008 Apr;82(4):834-48. doi: 10.1016/j.ajhg.2008.01.014. Epub 2008 Mar 27.

7.

Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy.

Singh NN, Lee BM, DiDonato CJ, Singh RN.

Future Med Chem. 2015;7(13):1793-808. doi: 10.4155/fmc.15.101. Epub 2015 Sep 18. Review.

8.

Oxidative Stress Triggers Body-Wide Skipping of Multiple Exons of the Spinal Muscular Atrophy Gene.

Seo J, Singh NN, Ottesen EW, Sivanesan S, Shishimorova M, Singh RN.

PLoS One. 2016 Apr 25;11(4):e0154390. doi: 10.1371/journal.pone.0154390. eCollection 2016.

9.

Alternative splicing in spinal muscular atrophy underscores the role of an intron definition model.

Singh NN, Singh RN.

RNA Biol. 2011 Jul-Aug;8(4):600-6. doi: 10.4161/rna.8.4.16224. Epub 2011 Jul 1.

10.

An antisense microwalk reveals critical role of an intronic position linked to a unique long-distance interaction in pre-mRNA splicing.

Singh NN, Hollinger K, Bhattacharya D, Singh RN.

RNA. 2010 Jun;16(6):1167-81. doi: 10.1261/rna.2154310. Epub 2010 Apr 22.

11.

hnRNP M facilitates exon 7 inclusion of SMN2 pre-mRNA in spinal muscular atrophy by targeting an enhancer on exon 7.

Cho S, Moon H, Loh TJ, Oh HK, Cho S, Choy HE, Song WK, Chun JS, Zheng X, Shen H.

Biochim Biophys Acta. 2014;1839(4):306-15. doi: 10.1016/j.bbagrm.2014.02.006. Epub 2014 Feb 15.

PMID:
24533984
12.

Securinine enhances SMN2 exon 7 inclusion in spinal muscular atrophy cells.

Chen YC, Chang JG, Liu TY, Jong YJ, Cheng WL, Yuo CY.

Biomed Pharmacother. 2017 Apr;88:708-714. doi: 10.1016/j.biopha.2017.01.104. Epub 2017 Jan 30.

PMID:
28152480
13.

Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 and SMN2.

Cartegni L, Hastings ML, Calarco JA, de Stanchina E, Krainer AR.

Am J Hum Genet. 2006 Jan;78(1):63-77. Epub 2005 Nov 16.

14.

Antisense oligonucleotide mediated therapy of spinal muscular atrophy.

Sivanesan S, Howell MD, Didonato CJ, Singh RN.

Transl Neurosci. 2013 Mar;4(1). doi: 10.2478/s13380-013-0109-2.

15.

Tetracyclines that promote SMN2 exon 7 splicing as therapeutics for spinal muscular atrophy.

Hastings ML, Berniac J, Liu YH, Abato P, Jodelka FM, Barthel L, Kumar S, Dudley C, Nelson M, Larson K, Edmonds J, Bowser T, Draper M, Higgins P, Krainer AR.

Sci Transl Med. 2009 Nov 4;1(5):5ra12. doi: 10.1126/scitranslmed.3000208.

16.

Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy.

Mitrpant C, Porensky P, Zhou H, Price L, Muntoni F, Fletcher S, Wilton SD, Burghes AH.

PLoS One. 2013 Apr 22;8(4):e62114. doi: 10.1371/journal.pone.0062114. Print 2013.

17.

A humanized Smn gene containing the SMN2 nucleotide alteration in exon 7 mimics SMN2 splicing and the SMA disease phenotype.

Gladman JT, Bebee TW, Edwards C, Wang X, Sahenk Z, Rich MM, Chandler DS.

Hum Mol Genet. 2010 Nov 1;19(21):4239-52. doi: 10.1093/hmg/ddq343. Epub 2010 Aug 12.

18.

HnRNP C1/C2 may regulate exon 7 splicing in the spinal muscular atrophy gene SMN1.

Irimura S, Kitamura K, Kato N, Saiki K, Takeuchi A, Gunadi, Matsuo M, Nishio H, Lee MJ.

Kobe J Med Sci. 2009 Mar 10;54(5):E227-36.

19.

An intronic structure enabled by a long-distance interaction serves as a novel target for splicing correction in spinal muscular atrophy.

Singh NN, Lawler MN, Ottesen EW, Upreti D, Kaczynski JR, Singh RN.

Nucleic Acids Res. 2013 Sep;41(17):8144-65. doi: 10.1093/nar/gkt609. Epub 2013 Jul 16.

20.

Antisense oligonucleotides and spinal muscular atrophy: skipping along.

Burghes AH, McGovern VL.

Genes Dev. 2010 Aug 1;24(15):1574-9. doi: 10.1101/gad.1961710.

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