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. Author manuscript; available in PMC: 2019 Sep 29.
Published in final edited form as: Bioconjug Chem. 2019 Jan 29;30(2):366–383. doi: 10.1021/acs.bioconjchem.8b00761

Figure 1.

Figure 1.

Mechanisms of antisense oligonucleotides. Normal expression of the gene and protein in the absence of ASO (1). After internalization, ASO hybridize to mRNA via complementary sequence pairing. The ASO/mRNA heteroduplex induces activation of RNase H and degradation of the mRNA (2) or ribosome blockage by steric hindrance (3). Both mechanisms result in inhibition of target protein production. Alternatively, penetration of ASO into the nucleus may lead to regulation of mRNA via inhibition of capping formation at the 5′ end (4), inhibition of splicing (5), or the activation of RNase H (6). Reproduced with permission from Chan, J. H. P., Lim, S., and Wong, W. S. F. (2006) Antisense Oligonucleotides: From Design to Therapeutic Application. Clin. Exp. Pharmacol. Physiol. 33, 533–540.12 Copyright 2006, Blackwell Publishing Asia Pty Lt.