Abstract:
:While disruption of alternative splicing underlies many diseases, modulation of splicing using antisense oligonucleotides (AONs) can have therapeutic implications. The most notable example is Duchenne muscular dystrophy (DMD), where antisense-mediated exon skipping can restore the open reading frame and allow the synthesis of partly functional dystrophin proteins instead of non-functional ones. This approach is currently tested in early phase clinical trials. In this review the development of the exon skipping approach in patient-derived cell cultures, animal models and patients is described and hurdles that have to be overcome to make this personalized medicine type approach widely applicable are discussed.
journal_name
RNA Bioljournal_title
RNA biologyauthors
Aartsma-Rus Adoi
10.4161/rna.7.4.12264subject
Has Abstractpub_date
2010-07-01 00:00:00pages
453-61issue
4eissn
1547-6286issn
1555-8584pii
12264journal_volume
7pub_type
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