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545 related items for PubMed ID: 26931466
1. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy. Zhao X, Feng Z, Ling KK, Mollin A, Sheedy J, Yeh S, Petruska J, Narasimhan J, Dakka A, Welch EM, Karp G, Chen KS, Metzger F, Ratni H, Lotti F, Tisdale S, Naryshkin NA, Pellizzoni L, Paushkin S, Ko CP, Weetall M. Hum Mol Genet; 2016 May 15; 25(10):1885-1899. PubMed ID: 26931466 [Abstract] [Full Text] [Related]
4. The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy. Kletzl H, Marquet A, Günther A, Tang W, Heuberger J, Groeneveld GJ, Birkhoff W, Mercuri E, Lochmüller H, Wood C, Fischer D, Gerlach I, Heinig K, Bugawan T, Dziadek S, Kinch R, Czech C, Khwaja O. Neuromuscul Disord; 2019 Jan 15; 29(1):21-29. PubMed ID: 30553700 [Abstract] [Full Text] [Related]
6. Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy. Williams JH, Schray RC, Patterson CA, Ayitey SO, Tallent MK, Lutz GJ. J Neurosci; 2009 Jun 17; 29(24):7633-8. PubMed ID: 19535574 [Abstract] [Full Text] [Related]
10. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy. Ratni H, Karp GM, Weetall M, Naryshkin NA, Paushkin SV, Chen KS, McCarthy KD, Qi H, Turpoff A, Woll MG, Zhang X, Zhang N, Yang T, Dakka A, Vazirani P, Zhao X, Pinard E, Green L, David-Pierson P, Tuerck D, Poirier A, Muster W, Kirchner S, Mueller L, Gerlach I, Metzger F. J Med Chem; 2016 Jul 14; 59(13):6086-100. PubMed ID: 27299419 [Abstract] [Full Text] [Related]
11. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Poirier A, Weetall M, Heinig K, Bucheli F, Schoenlein K, Alsenz J, Bassett S, Ullah M, Senn C, Ratni H, Naryshkin N, Paushkin S, Mueller L. Pharmacol Res Perspect; 2018 Dec 14; 6(6):e00447. PubMed ID: 30519476 [Abstract] [Full Text] [Related]
12. Improvement of spinal muscular atrophy via correction of the SMN2 splicing defect by Brucea javanica (L.) Merr. extract and Bruceine D. Baek J, Jeong H, Ham Y, Jo YH, Choi M, Kang M, Son B, Choi S, Ryu HW, Kim J, Shen H, Sydara K, Lee SW, Kim SY, Han SB, Oh SR, Cho S. Phytomedicine; 2019 Dec 14; 65():153089. PubMed ID: 31563042 [Abstract] [Full Text] [Related]
13. SMN deficiency alters Nrxn2 expression and splicing in zebrafish and mouse models of spinal muscular atrophy. See K, Yadav P, Giegerich M, Cheong PS, Graf M, Vyas H, Lee SG, Mathavan S, Fischer U, Sendtner M, Winkler C. Hum Mol Genet; 2014 Apr 01; 23(7):1754-70. PubMed ID: 24218366 [Abstract] [Full Text] [Related]
16. Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy. Woll MG, Qi H, Turpoff A, Zhang N, Zhang X, Chen G, Li C, Huang S, Yang T, Moon YC, Lee CS, Choi S, Almstead NG, Naryshkin NA, Dakka A, Narasimhan J, Gabbeta V, Welch E, Zhao X, Risher N, Sheedy J, Weetall M, Karp GM. J Med Chem; 2016 Jul 14; 59(13):6070-85. PubMed ID: 27299569 [Abstract] [Full Text] [Related]
18. Temporal and tissue-specific variability of SMN protein levels in mouse models of spinal muscular atrophy. Groen EJN, Perenthaler E, Courtney NL, Jordan CY, Shorrock HK, van der Hoorn D, Huang YT, Murray LM, Viero G, Gillingwater TH. Hum Mol Genet; 2018 Aug 15; 27(16):2851-2862. PubMed ID: 29790918 [Abstract] [Full Text] [Related]
19. 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 15; 153(2):264-275. PubMed ID: 31811660 [Abstract] [Full Text] [Related]
20. 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 01; 19(21):4239-52. PubMed ID: 20705738 [Abstract] [Full Text] [Related] Page: [Next] [New Search]