Romancing the spliceosome to fight spinal muscular atrophy

N Engl J Med. 2014 Oct 30;371(18):1752-4. doi: 10.1056/NEJMcibr1409795.
No abstract available

MeSH terms

  • Alternative Splicing / drug effects*
  • Animals
  • Cells, Cultured
  • Humans
  • Mice
  • Muscular Atrophy, Spinal / drug therapy*
  • Muscular Atrophy, Spinal / genetics
  • RNA, Messenger / metabolism
  • Small Molecule Libraries / administration & dosage*
  • Spliceosomes / metabolism
  • Survival of Motor Neuron 1 Protein / genetics
  • Survival of Motor Neuron 1 Protein / metabolism
  • Survival of Motor Neuron 2 Protein / genetics*
  • Survival of Motor Neuron 2 Protein / metabolism

Substances

  • RNA, Messenger
  • Small Molecule Libraries
  • Survival of Motor Neuron 1 Protein
  • Survival of Motor Neuron 2 Protein