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Article

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy

1 Jan 2011

Abstract excerpt

Antisense oligonucleotides are short nucleic acids designed to bind to specific messenger RNAs in order to modulate splicing patterns or inhibit protein translation. As such, they represent promising therapeutic tools for many disorders and have been actively developed for more than 20 years as a form of molecular medicine. Although significant progress has been made in developing these agents as drugs, they are...

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PMID: 21798085
DOI: 10.1186/2044-5040-1-8

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