RNA, Untranslated
- Name
- RNA, Untranslated
- Accession Number
- DBCAT002971
- Description
RNA which does not code for protein but has some enzymatic, structural or regulatory function. Although ribosomal RNA (RNA, RIBOSOMAL) and transfer RNA (RNA, TRANSFER) are also untranslated RNAs they are not included in this scope.
- Drugs
Drug Drug Description Inclisiran A PCSK9-targeting short interfering RNA (siRNA) that lowers plasma LDL-cholesterol levels. 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). Lumasiran Lumasiran is an interfering RNA that silences hydroxyacid oxidase 1 for the treatment of primary hypoxaluria type 1. 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)). NTLA-2001 NTLA-2001 is a clustered regularly interspaced short palindromic repeats CRISPR/ Cas9-based gene therapy consisting of a single guide RNA targeting the human TTR gene and a messenger RNA encoding Cas9. - Drugs & Drug Targets