Antisense Oligonucleotides

Antisense Oligonucleotides
Accession Number

Short fragments of DNA or RNA that are used to alter the function of target RNAs or DNAs to which they hybridize.

DrugDrug Description
MipomersenAn oligonucleotide drug used for the treatment of homozygous familial hypercholesterolemia.
CustirsenInvestigated for use/treatment in brain cancer and breast cancer.
FomivirsenIndicated for the local treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency syndrome (AIDS), when other therapy has been ineffective or is considered unsuitable [FDA Label, L1428].
OblimersenNot Annotated
EteplirsenAn antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
NusinersenA medication used to treat spinal muscular atrophy.
InotersenAn antisense oligonucleotide used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis.
GivosiranA 5-aminolevulinic acid synthase-directed small interfering RNA (siRNA) used in the prophylaxis of acute hepatic porphyria.
GolodirsenA drug used to treat certain mutations that cause Duchenne muscular dystrophy (DMD).
ViltolarsenAn antisense phosphorodiamidate morpholino oligonucleotide specific for exon 53 of the human DMD gene that is capable of inducing exon 53 skipping to produce a functional truncated dystrophin protein in Duchenne muscular dystrophy patients with specific underlying mutations.
CasimersenAn antisense phosphorodiamidate morpholino oligonucleotide used to treat Duchenne muscular dystrophy patients with mutations amenable to exon 45 skipping.
VutrisiranA transthyretin-directed small interfering RNA used to treat polyneuropathy associated with hereditary transthyretin-mediated amyloidosis.
TofersenAn antisense oligonucleotide indicated for the treatment of adult amyotrophic lateral sclerosis caused by SOD1 mutation.
AtesidorsenNot Annotated
Drugs & Drug Targets
MipomersenmRNA of ApoB-100target
CustirsenEstrogen receptor alphatarget
CustirsenNuclear factor NF-kappa-B p100 subunittarget
CustirsenNuclear factor NF-kappa-B p105 subunittarget
Fomivirsen30 kDa immediate-early protein 2target
Fomivirsen45 kDa immediate-early protein 2target
EteplirsenDMD-001 gene (exon 51 target site)target
NusinersenSurvival motor neuron proteintarget
InotersenTransthyretin mRNAtarget
GivosiranALAS1 mRNAtarget
ViltolarsenDMD gene (exon 53 viltolarsen target site)target
CasimersenDMD gene (exon 45 casimersen target site)target
CasimersenCytochrome P450 3A4enzyme
CasimersenCytochrome P450 3A5enzyme
CasimersenCytochrome P450 2C9enzyme
CasimersenCytochrome P450 2C19enzyme
VutrisiranTransthyretin mRNAtarget
VutrisiranSerum albumincarrier
TofersenSuperoxide dismutase 1 (SOD1)target