Nature Communications (Nov 2017)

Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers

  • Manaswini Sivaramakrishnan,
  • Kathleen D. McCarthy,
  • Sébastien Campagne,
  • Sylwia Huber,
  • Sonja Meier,
  • Angélique Augustin,
  • Tobias Heckel,
  • Hélène Meistermann,
  • Melanie N. Hug,
  • Pascale Birrer,
  • Ahmed Moursy,
  • Sarah Khawaja,
  • Roland Schmucki,
  • Nikos Berntenis,
  • Nicolas Giroud,
  • Sabrina Golling,
  • Manuel Tzouros,
  • Balazs Banfai,
  • Gonzalo Duran-Pacheco,
  • Jens Lamerz,
  • Ying Hsiu Liu,
  • Thomas Luebbers,
  • Hasane Ratni,
  • Martin Ebeling,
  • Antoine Cléry,
  • Sergey Paushkin,
  • Adrian R. Krainer,
  • Frédéric H.-T. Allain,
  • Friedrich Metzger

DOI
https://doi.org/10.1038/s41467-017-01559-4
Journal volume & issue
Vol. 8, no. 1
pp. 1 – 13

Abstract

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Small molecules correcting the splicing deficit of the survival of motor neuron 2 (SMN2) gene have been identified as having therapeutic potential. Here, the authors provide evidence that SMN2 mRNA forms a ribonucleoprotein complex that can be specifically targeted by these small molecules.