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Items: 15

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.

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.

3.

Antisense oligonucleotides for the treatment of spinal muscular atrophy.

Porensky PN, Burghes AH.

Hum Gene Ther. 2013 May;24(5):489-98. doi: 10.1089/hum.2012.225. Review.

4.

Spinal muscular atrophy: an update on therapeutic progress.

Seo J, Howell MD, Singh NN, Singh RN.

Biochim Biophys Acta. 2013 Dec;1832(12):2180-90. doi: 10.1016/j.bbadis.2013.08.005. Epub 2013 Aug 27. Review.

5.

Advances in therapeutic development for spinal muscular atrophy.

Howell MD, Singh NN, Singh RN.

Future Med Chem. 2014 Jun;6(9):1081-99. doi: 10.4155/fmc.14.63. Review.

6.

Therapeutics that directly increase SMN expression to treat spinal muscular atrophy.

Shababi M, Mattis VB, Lorson CL.

Drug News Perspect. 2010 Oct;23(8):475-82. doi: 10.1358/dnp.2010.23.8.1507295. Review.

PMID:
21031163
7.

[The role of RNA splicing in the pathogenesis of spinal muscular atrophy and development of its therapeutics].

Sahashi K, Sobue G.

Brain Nerve. 2014 Dec;66(12):1471-80. doi: 10.11477/mf.1416200061. Review. Japanese.

PMID:
25475034
8.

Is RNA manipulation a viable therapy for spinal muscular atrophy?

Horne C, Young PJ.

J Neurol Sci. 2009 Dec 15;287(1-2):27-31. doi: 10.1016/j.jns.2009.08.055. Epub 2009 Sep 15. Review.

PMID:
19758605
9.

Splicing therapeutics in SMN2 and APOB.

Khoo B, Krainer AR.

Curr Opin Mol Ther. 2009 Apr;11(2):108-15. Review.

10.

Therapeutics development for spinal muscular atrophy.

Sumner CJ.

NeuroRx. 2006 Apr;3(2):235-45. Review.

11.

Repair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy.

Nlend Nlend R, Meyer K, Schümperli D.

RNA Biol. 2010 Jul-Aug;7(4):430-40. Epub 2010 Jul 1. Review.

12.

How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy.

Singh NN, Howell MD, Androphy EJ, Singh RN.

Gene Ther. 2017 May 9. doi: 10.1038/gt.2017.34. [Epub ahead of print] Review.

PMID:
28485722
13.

Nusinersen: First Global Approval.

Hoy SM.

Drugs. 2017 Mar;77(4):473-479. doi: 10.1007/s40265-017-0711-7. Review.

PMID:
28229309
14.

Synthetic Nucleic Acids and Treatment of Neurological Diseases.

Corey DR.

JAMA Neurol. 2016 Oct 1;73(10):1238-1242. doi: 10.1001/jamaneurol.2016.2089. Review.

PMID:
27479447
15.

Gene-targeting pharmaceuticals for single-gene disorders.

Beaudet AL, Meng L.

Hum Mol Genet. 2016 Apr 15;25(R1):R18-26. doi: 10.1093/hmg/ddv476. Epub 2015 Nov 30. Review.

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