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. 2020 May 31;12(6):1424. doi: 10.3390/cancers12061424

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© 2020 by the authors.

Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).

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Antisense oligonucleotides (ASOs) in clinical use. (A) Nusinersen binds to a splicing regulatory sequence called intronic splicing silencer-N1 (ISS-N1) within intron 7 of SMN2 pre-mRNA, and enhances the inclusion of exon 7, resulting in the production of SMN2 mRNA coding the full-length SMN protein. (B) Mipomersen binds to ApoB-100 mRNA and causes its degradation, mediated by ribonuclease H (RNaseH). SMN2: survival of motor neuron 2: centromeric; SMN: survival of motor neuron; ApoB-100: apolipoprotein B-100.