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New developments in exon skipping and splice modulation therapies for neuromuscular diseases

12 Mar 2014

Abstract excerpt

INTRODUCTION: Antisense oligonucleotide (AON) therapy is a form of treatment for genetic or infectious diseases using small, synthetic DNA-like molecules called AONs. Recent advances in the development of AONs that show improved stability and increased sequence specificity have led to clinical trials for several neuromuscular diseases. Impressive preclinical and clinical data are published regarding the usage of...

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PMID: 24620745
DOI: 10.1517/14712598.2014.896335

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