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Antisense-mediated splice intervention to treat human disease: the odyssey continues

2019-05-22

Abstract excerpt

Recent approvals of oligonucleotide analogue drugs to alter gene expression have been welcomed by patient communities but not universally supported. These compounds represent a class of drugs that are designed to target a specific gene transcript, and they include a number of chemical entities to evoke different antisense mechanisms, depending upon the disease aetiology. To date, oligonucleotide therapeutics that...

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Literature Corpus work
fd445670-6a01-5884-b520-5f509c24bb11
DOI
10.12688/f1000research.18466.1
Open publication

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Antisense-mediated splice intervention to treat human disease: the odyssey continuesDOI 10.12688/f1000research.18466.1
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