Targeting splicing in spinal muscular atrophy

Ann Neurol. 2008 Jan;63(1):3-6. doi: 10.1002/ana.21305.
No abstract available

Publication types

  • Editorial
  • Comment

MeSH terms

  • Amiloride / analogs & derivatives
  • Amiloride / pharmacology
  • Amiloride / therapeutic use
  • Cyclic AMP Response Element-Binding Protein / genetics*
  • Exons / genetics
  • Gene Expression Regulation / genetics
  • Genetic Predisposition to Disease / genetics*
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Humans
  • Muscular Atrophy, Spinal / genetics*
  • Muscular Atrophy, Spinal / therapy*
  • Nerve Tissue Proteins / genetics*
  • RNA Splicing / genetics*
  • RNA-Binding Proteins / drug effects
  • RNA-Binding Proteins / genetics*
  • SMN Complex Proteins
  • Serine-Arginine Splicing Factors

Substances

  • Cyclic AMP Response Element-Binding Protein
  • Nerve Tissue Proteins
  • RNA-Binding Proteins
  • SMN Complex Proteins
  • SRSF3 protein, human
  • Serine-Arginine Splicing Factors
  • Amiloride
  • ethylisopropylamiloride