Welcome to the splice age: Antisense oligonucleotide-mediated exon skipping gains wider applicability

Elizabeth M. McNally*, Eugene J. Wyatt

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

3 Scopus citations

Abstract

Exon skipping uses antisense oligonucleotides (ASOs) to alter transcript splicing for the purpose of rescuing or modulating protein expression. In this issue of the JCI, Lee and colleagues developed and evaluated an ASO-dependent method for treating certain molecularly defined diseases associated with alterations in lamin A/C (LMNA) splicing. Exon skipping by ASOs is gaining traction as a therapeutic strategy, and the use of ASOs is now being applied to bypass mutations and generate modified but functional proteins for an array of genetic disorders.

Original languageEnglish (US)
Pages (from-to)1236-1238
Number of pages3
JournalJournal of Clinical Investigation
Volume126
Issue number4
DOIs
StatePublished - Apr 1 2016

ASJC Scopus subject areas

  • Medicine(all)

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