US EditionEnhancing ASOs Efficacy by Slowing Down Intracellular Transport
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Enhancing ASOs Efficacy by Slowing Down Intracellular Transport
Anti-sense oligonucleotides (ASOs) are short, modified, single-stranded oligonucleotides—made from DNA or locked nucleic acid (LNA)—with enhanced stability, activity, and bioavailability. They associate with RNA through sequence complementarity and can modify the expression (and/or splicing patterns) of target RNAs. These promising therapeutics are currently used to treat rare genetic diseases. Such RNA-based therapies are already being used suc…
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