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      Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.

      1 , ,
      Nucleic acid therapeutics

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          Abstract

          Alternative splicing is an important regulator of the transcriptome. However, mutations may cause alteration of splicing patterns, which in turn leads to disease. During the past 10 years, exon skipping has been looked upon as a powerful tool for correction of missplicing in disease and progress has been made towards clinical trials. In this review, we discuss the use of antisense oligonucleotides to correct splicing defects through exon skipping, with a special focus on diseases affecting the nervous system, and the latest stage achieved in its progress.

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          Author and article information

          Journal
          Nucleic Acid Ther
          Nucleic acid therapeutics
          2159-3345
          2159-3337
          Feb 2014
          : 24
          : 1
          Affiliations
          [1 ] 1 Center for Integrative Biology (CIBIO), University of Trento , Trento, Italy .
          Article
          10.1089/nat.2013.0461
          24506781
          ff6b6502-ba10-4bb6-8d0f-b4d4b393e55c
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