Welcome to the splice age: antisense oligonucleotide-mediated exon skipping gains wider applicability.
Level V
Where this comes from
- Record sourced from PubMed, PMID 26999602.
- Also identified by DOI 10.1172/JCI86799 and PMC identifier 4811139.
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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.
Medical subject headings
- Alternative Splicing
- Lamin Type A
- Oligodeoxyribonucleotides, Antisense
- Progeria
- RNA, Messenger