Abstract. Here we discuss three RNA therapeutic technologies exploiting various oligonucleotides that bind RNA by base-pairing in a sequence-specific manner yet have different mechanisms of action and effects. RNA interference and antisense oligonucleotides downregulate gene expression by enzyme-dependent degradation of targeted mRNA.
Oligonucleotides, Antisense. locked nucleic acid. Locked nucleic acid (LNA) is the term for oligonucleotides that contain one or more nucleotide building blocks in which an extra methylene bridge fixes the ribose moiety either in the C3'-endo (beta-D-LNA) or C2'-endo (alpha-L-LNA) conformation. The beta-D-LNA modification results in significant Antisense mechanism (A) and phosphorothioate (B).(A) The use of LNA oligonucleotides as inhibitors of disease—forming proteins, or reduction of harmful RNA, is based on specific hybridization to

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Antisense oligonucleotides targeting multiple types of non-productive splicing events lead to increases in productive mRNA and protein in a dose-dependent manner in vitro. Moreover

To reveal the DNA targets of KCNQ1OT1, we performed chromatin isolation by ChIRP–seq 32,38 in HEK293T cells using the same antisense DNA oligonucleotides that were used for the ChOP described

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