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Tox considerations for oligonucleotide therapeutics
19-21 Induce mRNA degradation via RNAi pathway miRNA ssRNA 20-25 Inhibit translation via binding to 3’UTR and/or promote mRNA degradation Decoy dsDNA >20 Inhibit protein function through high affinity binding Binds to DNA binding site of transcription factor, inhibiting gene expression Aptamer ss or dsRNA, DNA 30-40 Binds to target protein ...
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