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.
Titolo: | Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases | |
Autori: | Siva, Kavitha; Covello, Giuseppina; Denti, Michela Alessandra | |
Autori Unitn: | ||
Titolo del periodico: | NUCLEIC ACID THERAPEUTICS | |
Anno di pubblicazione: | 2014 | |
Codice identificativo Scopus: | 2-s2.0-84893582109 | |
Codice identificativo WOS: | WOS:000331389100008 | |
Digital Object Identifier (DOI): | http://dx.doi.org/10.1089/nat.2013.0461 | |
Handle: | http://hdl.handle.net/11572/67315 | |
Appare nelle tipologie: | 03.1 Articolo su rivista (Journal article) |
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