Nature Communications (Apr 2016)

Therapeutic activity of modified U1 core spliceosomal particles

  • Malgorzata Ewa Rogalska,
  • Mojca Tajnik,
  • Danilo Licastro,
  • Erica Bussani,
  • Luca Camparini,
  • Chiara Mattioli,
  • Franco Pagani

DOI
https://doi.org/10.1038/ncomms11168
Journal volume & issue
Vol. 7, no. 1
pp. 1 – 13

Abstract

Read online

Modification of the spliceosome is being tested as a potential therapy for exon-skipping diseases, such as spinal muscular atrophy (SMA). Here the authors show that 70K and stem loop IV structural elements of a modified U1 particle are essential for splicing enhancement and effective treatment of SMA mice.