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5. 5-(N-ethyl-N-isopropyl)-amiloride enhances SMN2 exon 7 inclusion and protein expression in spinal muscular atrophy cells. Yuo CY; Lin HH; Chang YS; Yang WK; Chang JG Ann Neurol; 2008 Jan; 63(1):26-34. PubMed ID: 17924536 [TBL] [Abstract][Full Text] [Related]
6. Splicing of the Survival Motor Neuron genes and implications for treatment of SMA. Bebee TW; Gladman JT; Chandler DS Front Biosci (Landmark Ed); 2010 Jun; 15(3):1191-1204. PubMed ID: 20515750 [TBL] [Abstract][Full Text] [Related]
7. 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]
8. A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo. Dickson A; Osman E; Lorson CL Hum Gene Ther; 2008 Nov; 19(11):1307-15. PubMed ID: 19848583 [TBL] [Abstract][Full Text] [Related]
9. 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]
10. Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape. Wood MJA; Talbot K; Bowerman M Hum Mol Genet; 2017 Oct; 26(R2):R151-R159. PubMed ID: 28977438 [TBL] [Abstract][Full Text] [Related]
11. Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of Toosaranont J; Ruschadaariyachat S; Mujchariyakul W; Arora JK; Charoensawan V; Suktitipat B; Palmer TN; Fletcher S; Wilton SD; Mitrpant C Int J Mol Sci; 2022 Apr; 23(7):. PubMed ID: 35409296 [TBL] [Abstract][Full Text] [Related]
12. High expression level of Tra2-β1 is responsible for increased SMN2 exon 7 inclusion in the testis of SMA mice. Chen YC; Chang JG; Jong YJ; Liu TY; Yuo CY PLoS One; 2015; 10(3):e0120721. PubMed ID: 25781985 [TBL] [Abstract][Full Text] [Related]
13. Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model. Hua Y; Sahashi K; Rigo F; Hung G; Horev G; Bennett CF; Krainer AR Nature; 2011 Oct; 478(7367):123-6. PubMed ID: 21979052 [TBL] [Abstract][Full Text] [Related]
14. 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]
15. Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene. Pao PW; Wee KB; Yee WC; Pramono ZA Mol Ther; 2014 Apr; 22(4):854-61. PubMed ID: 24317636 [TBL] [Abstract][Full Text] [Related]
16. An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA. Zhang ML; Lorson CL; Androphy EJ; Zhou J Gene Ther; 2001 Oct; 8(20):1532-8. PubMed ID: 11704813 [TBL] [Abstract][Full Text] [Related]
17. Antisense oligonucleotides for the treatment of spinal muscular atrophy. Porensky PN; Burghes AH Hum Gene Ther; 2013 May; 24(5):489-98. PubMed ID: 23544870 [TBL] [Abstract][Full Text] [Related]
18. SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2 beta 1. Young PJ; DiDonato CJ; Hu D; Kothary R; Androphy EJ; Lorson CL Hum Mol Genet; 2002 Mar; 11(5):577-87. PubMed ID: 11875052 [TBL] [Abstract][Full Text] [Related]