Antisense therapy

Antisense therapy uses synthetic nucleic-acid strands to bind specific RNA molecules and alter gene expression or RNA processing.

RNase H: An enzyme that degrades the RNA strand of an RNA–DNA hybrid. Many antisense oligonucleotides recruit it to cleave their bound target RNA.
Nusinersen: An antisense oligonucleotide that alters SMN2 splicing to increase production of functional SMN protein. It treats spinal muscular atrophy by promoting inclusion of SMN2 exon 7.
Oligonucleotide: A short nucleic-acid polymer composed of linked nucleotide units. Antisense medicines are engineered oligonucleotides with defined sequences and chemical modifications.
RNA interference: A gene-silencing process in which small RNAs guide cellular complexes to complementary RNA targets. Unlike many antisense drugs, it relies on RNA-guided cellular machinery to silence targets.
Steric-blocking oligonucleotide: An oligonucleotide that changes RNA function by physically blocking access to a binding site. This mechanism can alter splicing or translation without degrading the target RNA.
Eteplirsen: A phosphorodiamidate morpholino oligonucleotide that promotes exon 51 skipping in dystrophin pre-mRNA. It enables a subset of Duchenne muscular dystrophy patients to produce a shorter dystrophin.
Watson–Crick base pairing: The complementary pairing of adenine with thymine or uracil and guanine with cytosine. Complementary base pairing lets an antisense strand recognize a chosen RNA sequence.
Small interfering RNA: A short double-stranded RNA that guides RNA-induced silencing complex to complementary transcripts. It is a therapeutic alternative that commonly uses a different silencing pathway and delivery profile.
Splice-switching oligonucleotide: An oligonucleotide that redirects pre-mRNA splicing by masking selected RNA sequences. It changes which exons enter mature RNA rather than simply reducing RNA abundance.
Inotersen: An antisense oligonucleotide that reduces transthyretin production by targeting its messenger RNA. It is used to treat hereditary transthyretin amyloidosis.

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NucleobaseRelated: Complementary bases enable antisense molecules to bind chosen RNA targets.