title: Targeting splicing in spinal muscular atrophy.
authors.forename: Barrington G
authors.initials: BG
authors.lastname: Burnett
authors.forename: Charlotte J
authors.initials: CJ
authors.lastname: Sumner
mesh_terms: D000584:Amiloride / Q000031:analogs & derivatives / Q000494:pharmacology / Q000627:therapeutic use; D017362:Cyclic AMP Response Element-Binding Protein / Q000235:genetics*; D005091:Exons / Q000235:genetics; D005786:Gene Expression Regulation / Q000235:genetics; D020022:Genetic Predisposition to Disease / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000235:genetics* / Q000628:therapy*; D009419:Nerve Tissue Proteins / Q000235:genetics*; D012326:RNA Splicing / Q000235:genetics*; D016601:RNA-Binding Proteins / Q000187:drug effects / Q000235:genetics*; D055532:SMN Complex Proteins; D000068103:Serine-Arginine Splicing Factors
chemical_list: D017362:Cyclic AMP Response Element-Binding Protein; D009419:Nerve Tissue Proteins; D016601:RNA-Binding Proteins; D055532:SMN Complex Proteins; C491388:SRSF3 protein, human; D000068103:Serine-Arginine Splicing Factors; D000584:Amiloride; C039614:ethylisopropylamiloride