- Oligonucleotides, Antisense
- Accession Number
Short fragments of DNA or RNA that are used to alter the function of target RNAs or DNAs to which they hybridize.
Drug Drug Description Mipomersen An oligonucleotide drug used for the treatment of homozygous familial hypercholesterolemia. Custirsen Investigated for use/treatment in brain cancer and breast cancer. Fomivirsen Indicated 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]. Oblimersen Not Annotated Eteplirsen An antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) only in patients who are confirmed to have a specific type of mutation. Nusinersen A medication used to treat spinal muscular atrophy. Inotersen An antisense oligonucleotide used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis. Givosiran A 5-aminolevulinic acid synthase-directed small interfering RNA (siRNA) used in the prophylaxis of acute hepatic porphyria. Golodirsen A drug used to treat certain mutations that cause Duchenne muscular dystrophy (DMD). Viltolarsen An 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.
- Drugs & Drug Targets