2024-11-04
Antisense Oligonucleotide ASO Design Essentials and Considerations
Traditional antisense oligonucleotide (ASO)-based gene regulation (including silencing or enhancement of gene expression) targets mRNAs and can act either in the nucleus or in the cytoplasm. In the nucleus (targeting pre-mRNA precursors), regulation usually acts by altering polyadenylation, altering splicing events, or cleaving inter-nucleotide bonds.