Antisense oligonucleotide

An antisense oligonucleotide is a short synthetic nucleic acid designed to bind a chosen RNA sequence and alter its activity, often by blocking translation or promoting RNA degradation.

Watson–Crick base pairing: The pairing of nucleic-acid bases through hydrogen bonds, with adenine pairing with thymine or uracil and guanine with cytosine. Complementary bases let an oligonucleotide recognize its chosen RNA sequence.
Oligonucleotide: A short chain of nucleotides joined by phosphodiester or related chemical linkages. Antisense agents are chemically modified oligonucleotides built to bind RNA.
Nusinersen: An antisense medicine that modifies SMN2 pre-mRNA splicing to increase production of functional SMN protein. It treats spinal muscular atrophy by promoting inclusion of SMN2 exon 7.
Small interfering RNA: A short double-stranded RNA that guides the RNA-induced silencing complex to complementary messenger RNA. It shares sequence-directed silencing with antisense drugs but depends on RISC-mediated cleavage.
Off-target effects: Unintended biological effects caused when a treatment interacts with targets other than its intended target. Partial sequence matches and chemistry-dependent interactions can alter unintended transcripts or proteins.
RNase H: An enzyme that cleaves the RNA strand of an RNA–DNA hybrid. Many DNA-like antisense oligonucleotides recruit RNase H to degrade target RNA.
RNA: A nucleic acid made from ribonucleotides that carries, regulates, or performs biological information. It is the molecular target whose sequence and structure guide antisense design.
Eteplirsen: An antisense oligonucleotide designed to skip exon 51 in dystrophin pre-mRNA. It illustrates mutation-specific exon skipping for some people with Duchenne muscular dystrophy.
Small-molecule drug: A low-molecular-weight compound that alters biological activity through interactions with molecular targets. Small molecules can reach targets inaccessible to oligonucleotides and follow different delivery constraints.
Innate immune response: The body's rapid, nonspecific defense response to infection, tissue damage, or foreign molecular patterns. Some oligonucleotide sequences or chemical features can activate immune sensors.

Linked from 7 pages