Antisense drugs are short, chemically modified, single-stranded nucleic acids (antisense oligonucleotides) that have the ability to target any gene product of interest.
Antisense oligonucleotides offer new opportunities for therapeutic intervention because they act inside the cell to influence protein production. They do this by targeting RNA to either prevent the production of disease-causing proteins, increase the production of proteins deficient in disease, or target toxic RNAs that are unable to generate proteins. Antisense drugs are also able to target emerging biology mediated by microRNAs and long, non-coding RNAs.
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