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Updated: May 3, 2026

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
Published on: May 11, 2018
Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases
Kavitha Siva1, Giuseppina Covello, Michela A Denti
11 Center for Integrative Biology (CIBIO), University of Trento , Trento, Italy .
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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