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3. Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron. Singh NK; Singh NN; Androphy EJ; Singh RN Mol Cell Biol; 2006 Feb; 26(4):1333-46. PubMed ID: 16449646 [TBL] [Abstract][Full Text] [Related]
4. ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy. Ottesen EW Transl Neurosci; 2017 Jan; 8():1-6. PubMed ID: 28400976 [TBL] [Abstract][Full Text] [Related]
5. Mechanism of Splicing Regulation of Spinal Muscular Atrophy Genes. Singh RN; Singh NN Adv Neurobiol; 2018; 20():31-61. PubMed ID: 29916015 [TBL] [Abstract][Full Text] [Related]
6. 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. PubMed ID: 23994186 [TBL] [Abstract][Full Text] [Related]
7. The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy. Singh RN; Ottesen EW; Singh NN Neurosci Insights; 2020; 15():2633105520973985. PubMed ID: 33283185 [TBL] [Abstract][Full Text] [Related]
8. 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 Sep; 24(9):520-526. PubMed ID: 28485722 [TBL] [Abstract][Full Text] [Related]
9. High Concentration of an ISS-N1-Targeting Antisense Oligonucleotide Causes Massive Perturbation of the Transcriptome. Ottesen EW; Luo D; Singh NN; Singh RN Int J Mol Sci; 2021 Aug; 22(16):. PubMed ID: 34445083 [TBL] [Abstract][Full Text] [Related]
10. RNA in spinal muscular atrophy: therapeutic implications of targeting. Singh RN; Seo J; Singh NN Expert Opin Ther Targets; 2020 Aug; 24(8):731-743. PubMed ID: 32538213 [TBL] [Abstract][Full Text] [Related]
11. Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy. Osman EY; Yen PF; Lorson CL Mol Ther; 2012 Jan; 20(1):119-26. PubMed ID: 22031236 [TBL] [Abstract][Full Text] [Related]
12. Structural Context of a Critical Exon of Spinal Muscular Atrophy Gene. Singh NN; O'Leary CA; Eich T; Moss WN; Singh RN Front Mol Biosci; 2022; 9():928581. PubMed ID: 35847983 [TBL] [Abstract][Full Text] [Related]
13. Combinatorial treatment for spinal muscular atrophy: An Editorial for 'Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells' on page 264. Poletti A; Fischbeck KH J Neurochem; 2020 Apr; 153(2):146-149. PubMed ID: 32056234 [TBL] [Abstract][Full Text] [Related]
14. 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. PubMed ID: 26381381 [TBL] [Abstract][Full Text] [Related]
15. Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy. Son HW; Yokota T Methods Mol Biol; 2018; 1828():57-68. PubMed ID: 30171534 [TBL] [Abstract][Full Text] [Related]
16. 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; 6(3):341-50. PubMed ID: 19430205 [TBL] [Abstract][Full Text] [Related]
17. LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts. Touznik A; Maruyama R; Hosoki K; Echigoya Y; Yokota T Sci Rep; 2017 Jun; 7(1):3672. PubMed ID: 28623256 [TBL] [Abstract][Full Text] [Related]
18. Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells. Pagliarini V; Guerra M; Di Rosa V; Compagnucci C; Sette C J Neurochem; 2020 Apr; 153(2):264-275. PubMed ID: 31811660 [TBL] [Abstract][Full Text] [Related]
19. High Concentration or Combined Treatment of Antisense Oligonucleotides for Spinal Muscular Atrophy Perturbed Wijaya YOS; Niba ETE; Nishio H; Okamoto K; Awano H; Saito T; Takeshima Y; Shinohara M Genes (Basel); 2022 Apr; 13(4):. PubMed ID: 35456491 [TBL] [Abstract][Full Text] [Related]
20. 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. PubMed ID: 25727246 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]