449 related articles for article (PubMed ID: 28389270)
21. Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy.
Thurmond J; Butchbach ME; Palomo M; Pease B; Rao M; Bedell L; Keyvan M; Pai G; Mishra R; Haraldsson M; Andresson T; Bragason G; Thosteinsdottir M; Bjornsson JM; Coovert DD; Burghes AH; Gurney ME; Singh J
J Med Chem; 2008 Feb; 51(3):449-69. PubMed ID: 18205293
[TBL] [Abstract][Full Text] [Related]
22. 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. PubMed ID: 28152480
[TBL] [Abstract][Full Text] [Related]
23. Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy.
Ando S; Suzuki S; Okubo S; Ohuchi K; Takahashi K; Nakamura S; Shimazawa M; Fuji K; Hara H
Sci Rep; 2020 Oct; 10(1):17472. PubMed ID: 33060681
[TBL] [Abstract][Full Text] [Related]
24. 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; 29(24):7633-8. PubMed ID: 19535574
[TBL] [Abstract][Full Text] [Related]
25. Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.
Osman EY; Washington CW; Kaifer KA; Mazzasette C; Patitucci TN; Florea KM; Simon ME; Ko CP; Ebert AD; Lorson CL
Mol Ther; 2016 Sep; 24(9):1592-601. PubMed ID: 27401142
[TBL] [Abstract][Full Text] [Related]
26. Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy.
Shababi M; Glascock J; Lorson CL
Hum Gene Ther; 2011 Feb; 22(2):135-44. PubMed ID: 20804424
[TBL] [Abstract][Full Text] [Related]
27. Spinal muscular atrophy: from gene to therapy.
Wirth B; Brichta L; Hahnen E
Semin Pediatr Neurol; 2006 Jun; 13(2):121-31. PubMed ID: 17027862
[TBL] [Abstract][Full Text] [Related]
28. Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.
Nizzardo M; Simone C; Salani S; Ruepp MD; Rizzo F; Ruggieri M; Zanetta C; Brajkovic S; Moulton HM; Müehlemann O; Bresolin N; Comi GP; Corti S
Clin Ther; 2014 Mar; 36(3):340-56.e5. PubMed ID: 24636820
[TBL] [Abstract][Full Text] [Related]
29. 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. PubMed ID: 23185376
[TBL] [Abstract][Full Text] [Related]
30. VPA response in SMA is suppressed by the fatty acid translocase CD36.
Garbes L; Heesen L; Hölker I; Bauer T; Schreml J; Zimmermann K; Thoenes M; Walter M; Dimos J; Peitz M; Brüstle O; Heller R; Wirth B
Hum Mol Genet; 2013 Jan; 22(2):398-407. PubMed ID: 23077215
[TBL] [Abstract][Full Text] [Related]
31. Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.
Nizzardo M; Simone C; Dametti S; Salani S; Ulzi G; Pagliarani S; Rizzo F; Frattini E; Pagani F; Bresolin N; Comi G; Corti S
Sci Rep; 2015 Jun; 5():11746. PubMed ID: 26123042
[TBL] [Abstract][Full Text] [Related]
32. Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs.
Madocsai C; Lim SR; Geib T; Lam BJ; Hertel KJ
Mol Ther; 2005 Dec; 12(6):1013-22. PubMed ID: 16226920
[TBL] [Abstract][Full Text] [Related]
33. 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]
34. 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; 29(1):21-29. PubMed ID: 30553700
[TBL] [Abstract][Full Text] [Related]
35. Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts.
Mattis VB; Rai R; Wang J; Chang CW; Coady T; Lorson CL
Hum Genet; 2006 Nov; 120(4):589-601. PubMed ID: 16951947
[TBL] [Abstract][Full Text] [Related]
36. 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]
37. A SMN2 Splicing Modifier Rescues the Disease Phenotypes in an In Vitro Human Spinal Muscular Atrophy Model.
Son YS; Choi K; Lee H; Kwon O; Jung KB; Cho S; Baek J; Son B; Kang SM; Kang M; Yoon J; Shen H; Lee S; Oh JH; Lee HA; Lee MO; Cho HS; Jung CR; Kim J; Cho S; Son MY
Stem Cells Dev; 2019 Apr; 28(7):438-453. PubMed ID: 30667343
[TBL] [Abstract][Full Text] [Related]
38. Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts.
Skordis LA; Dunckley MG; Yue B; Eperon IC; Muntoni F
Proc Natl Acad Sci U S A; 2003 Apr; 100(7):4114-9. PubMed ID: 12642665
[TBL] [Abstract][Full Text] [Related]
39. 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; 1(5):5ra12. PubMed ID: 20161659
[TBL] [Abstract][Full Text] [Related]
40. 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]
[Previous] [Next] [New Search]
