DescriptionAntisense Oligonucleotide Use in ncRNA therapy.png
English: ASO (Antisense oligonucleotide) are indicated as blue single stranded molecules in the cytoplasm. Double stranded siRNAs are indicated as a blue and red strand incorporated together. 1. Once they enter the nucleus, they undergo further modifications allowing them to bind to their target (lncRNA or mRNA) and degrade them using RNase H.The degradation of mRNA and lncRNA can be done through the use of siRNA in conjunction with RISC complexes found inside the cytoplasm or nucleus. ASOs can also be used to preferentially modulate processing of mRNA and lncRNA transcripts. The ASO has the ability to bind to pre-mRNA structure and directly modulate splicing (5), by masking splicing enhancer and repressors sequences, causing skipping in exons (2). It also has the ability to modulate polyadenylation selection in those transcripts with more than one polyA site in the 3'-UTR (6). We can also have the ASO binding to mRNA and sterically blocking the ribosomal subunits from translation into proteins (3), this can also be done using siRNA. In order to target ncRNAs within the nucleus we can also make use of viruses, which can be used to knockout (short RNA) shRNA or lncRNA (4), same vectors can also be used to upregulate lncRNA activity.
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