RNA, Antisense
- Name
- RNA, Antisense
- Accession Number
- DBCAT002973
- Description
RNA molecules which hybridize to complementary sequences in either RNA or DNA altering the function of the latter. Endogenous antisense RNAs function as regulators of gene expression by a variety of mechanisms. Synthetic antisense RNAs are used to effect the functioning of specific genes for investigative or therapeutic purposes.
- Drugs
Drug Drug Description Inclisiran A PCSK9-targeting short interfering RNA (siRNA) that lowers plasma LDL-cholesterol levels. Givosiran A 5-aminolevulinic acid synthase-directed small interfering RNA (siRNA) used in the prophylaxis of acute hepatic porphyria. Lumasiran Lumasiran is an interfering RNA that silences hydroxyacid oxidase 1 for the treatment of primary hypoxaluria type 1. Bevasiranib Investigated for use/treatment in macular degeneration and diabetic retinopathy. Patisiran A transthyretin-directed small interfering RNA used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis. Fitusiran Fitusiran is under investigation in clinical trial NCT02554773 (An Open-label Extension Study of an Investigational Drug, ALN-AT3SC, in Patients With Moderate or Severe Hemophilia A or B). Revusiran Revusiran is under investigation in clinical trial NCT02319005 (ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)). Vutrisiran A transthyretin-directed small interfering RNA used to treat polyneuropathy associated with hereditary transthyretin-mediated amyloidosis. - Drugs & Drug Targets
Drug Target Type Inclisiran Proprotein convertase subtilisin/kexin type 9 target Givosiran ALAS1 mRNA target Lumasiran Hydroxyacid oxidase 1 target Lumasiran Serum albumin carrier Lumasiran Cytochrome P450 2C8 enzyme Bevasiranib Vascular endothelial growth factor A target Patisiran Transthyretin mRNA target Patisiran Serum albumin carrier Patisiran alpha1-acid glycoprotein carrier Revusiran Transthyretin target Vutrisiran Transthyretin mRNA target Vutrisiran Serum albumin carrier