RNA, Small Untranslated
- Name
- RNA, Small Untranslated
- Accession Number
- DBCAT002970
- Description
Short RNA, about 200 base pairs in length or shorter, that does not code for protein.
- 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