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Antisense molecules: A new class of drugs

      An improved understanding of disease pathogenesis leads to identification of novel therapeutic targets. From a pharmacologic point of view, these can be addressed by small chemical compounds, so-called biologicals (eg, mAbs and recombinant proteins), or by a rather new class of molecule based on the antisense concept. Recently, a new wave of clinical studies exploring antisense strategies is evolving. In addition to cancer, they include predominantly trials on infectious and noninfectious diseases, such as chronic inflammatory and metabolic conditions. This article, based on a systematic PubMed literature search, highlights recent developments in this emerging field.

      Key words

      Abbreviations used:

      ALS (Amylotrophic lateral sclerosis), AMD (Age-related macular degeneration), CMV (Cytomegalovirus), CNV (Choroidal neovascularization), CVD (Cardiovascular disease), DMD (Duchenne muscular dystrophy), FDA (US Food and Drug Administration), HCV (Hepatitis C virus), IRS-1 (Insulin receptor substrate 1), LDL-C (Low-density lipoprotein cholesterol), MOE (2′-O-methoxyethyl), ODN (Antisense oligodeoxyribonucleotide), PCSK9 (Proprotein convertase subtilisin/kexin type 9), PT (Phosphorothioate), siRNA (Small interfering RNA), TLR (Toll-like receptor), VEGF (Vascular endothelial growth factor)
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