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Article

Development of Therapeutic Splice-Switching Oligonucleotides

13 May 2014

Abstract excerpt

Synthetic splice-switching oligonucleotides (SSOs) target nuclear pre-mRNA molecules to change exon splicing and generate an alternative protein isoform. Clinical trials with two competitive SSO drugs are underway to treat Duchenne muscular dystrophy (DMD). Beyond DMD, many additional therapeutic applications are possible, with some in phase 1 clinical trials or advanced preclinical evaluation. Here, we present...

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Share to Neurogenetic and Muscular Disorders Research
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PMID: 24826963
DOI: 10.1089/hum.2013.234

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