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

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.

SUBMITTER: Siva K

PROVIDER: S-EPMC3922311 | biostudies-literature | 2014 Feb

REPOSITORIES: biostudies-literature

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

Siva Kavitha K Covello Giuseppina G Denti Michela A MA

Nucleic acid therapeutics 20140201 1

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 n ...[more]

PMID: 24506781

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Exon-skipping antisense oligonucleotides for cystic fibrosis therapy.

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Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy.

Project description:[This corrects the article DOI: 10.1371/journal.pone.0111079.].

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Correction: Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy.

Project description:[This corrects the article DOI: 10.1371/journal.pone.0111079.].

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A prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene.

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