Antisense oligonucleotide (ASO)
A short single strand designed to bind a target messenger RNA and block or destroy it.
An antisense oligonucleotide is a short, single stranded piece of chemically modified DNA or RNA designed to be complementary to a specific messenger RNA. When it binds its target, it can either recruit the enzyme RNase H to cut the message, or block the way the message is processed and translated.
ASOs are one of the most clinically validated oligonucleotide modalities, with approved drugs across neuromuscular, cardiovascular and metabolic disease.
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