Antisense therapy

Definition

Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. Several ASOs have been approved in the United States, the European Union, and elsewhere.

Related concepts

ATXN2 Acromegaly Active substance Age related macular degenartion Akcea Therapeutics Alexander disease Alnylam Pharmaceuticals Alzheimer's disease Amyotrophic lateral sclerosis Angelman syndrome Antisense Antisense mRNA Antisense oligonucleotide Batten disease Beta thalassemia Biogen Blood brain barrier Cancer Cardiovascular disease Centronuclear myopathy Cerebrospinal fluid Clinical trial phase Coagulopathy Cystic fibrosis Cytomegalovirus retinitis DRPLA Diabetes Duchenne muscular dystrophy Epidermolysis bullosa dystrophica Eteplirsen European Medicines Agency Exon FDA FUS (gene)Familial Chylomicronemia Syndrome Familial chylomicronaemia syndrome Familial hypercholesterolemia Fomivirsen Food and Drug Administration Frontotemporal dementia Fuchs' dystrophy Golodirsen Hepatitis B Hereditary angioedema Hereditary transthyretin-mediated amyloidosis Hoffmann-La Roche Huntington's disease Hypertension IgA nephropathy Inotersen Intrathecal administration Ionis Pharmaceuticals Kjer's optic neuropathy Lafora disease Leber's hereditary optic neuropathy Lipoprotein(a)Locked nucleic acid Lymphoma Messenger RNA Mipomersen Morpholino Multiple myeloma Multiple system atrophy Myotonic dystrophy N-Acetylgalactosamine Neuraxis Non-alcoholic fatty liver disease Nuclease Nucleic acid Nusinersen Oligonucleotide Oligonucleotide synthesis Orphan drug Parkinson's disease Patisiran Pelizaeus–Merzbacher disease Peptide nucleic acid Personalized medicine Prion disease Prostate cancer Public domain RNA interference Retinitis pigmentosa Rett syndrome Ribonuclease H SOD1 STAT3 Small interfering RNA Spinal muscular atrophy Spinocerebellar Ataxia Type-3 Splicing (genetics)Stargardt disease The New York Times Tofersen Transthyretin Usher syndrome Viltolarsen Volanesorsen

5 concepts already in your glossary