Open AccessExon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases
Author(s) -
Kavitha Siva,
Giuseppina Covello,
Michela Alessandra Denti
Publication year - 2014
Publication title -
nucleic acid therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.255
H-Index - 67
eISSN - 2159-3345
pISSN - 2159-3337
DOI - 10.1089/nat.2013.0461
Subject(s) - exon skipping , exon , rna splicing , alternative splicing , biology , transcriptome , oligonucleotide , genetics , computational biology , bioinformatics , gene , gene expression , rna
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.