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Showing 1–2 of 2 results
Advanced filters: Author: Malgorzata Ewa Rogalska Clear advanced filters

  • 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.

    • Malgorzata Ewa Rogalska
    • Mojca Tajnik
    • Franco Pagani
    ResearchOpen Access
    Nature Communications
    Volume: 7, P: 1-13

  • Alternative splicing of pre-mRNAs is key for cellular function and underpins the aetiology of numerous diseases. Here, we review major advances in understanding the structures and functions of the splicing machinery and its regulation, and in harnessing this knowledge for the design of novel therapies.

    • Malgorzata Ewa Rogalska
    • Claudia Vivori
    • Juan Valcárcel
    Reviews
    Nature Reviews Genetics
    Volume: 24, P: 251-269