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Antisense oligonucleotide (ASO)

Definition
AI-generated

An antisense oligonucleotide (ASO) is a short synthetic nucleic acid designed to be complementary to a target RNA (pre-mRNA or mature transcript).

Synonyms
Plural
ASOs; antisense oligonucleotides

Why it matters in GWAS

Functional follow-up of regulatory or coding hits sometimes uses ASO-mediated knockdown or splice switching in models; understanding ASO mechanism helps connect variant-to-gene hypotheses to manipulable RNA targets and therapeutic analogues.

Example usage

"An ASO targeting the risk haplotype-associated isoform recapitulated the splicing defect seen in patient fibroblasts."

References

  • Crooke ST, et al. (2018). Antisense technology: an overview and prospectus. Nat Rev Drug Discov. https://doi.org/10.1038/nrd.2018.148
  • Lodish H, et al. (2021). Molecular Cell Biology. 9th ed. W. H. Freeman.

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