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Making sense of antisense oligonucleotide therapy
Author(s) -
Greener Mark
Publication year - 2020
Publication title -
prescriber
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.106
H-Index - 3
eISSN - 1931-2253
pISSN - 0959-6682
DOI - 10.1002/psb.1848
Subject(s) - spinal muscular atrophy , medicine , antisense therapy , duchenne muscular dystrophy , oligonucleotide , neuromuscular disease , muscular dystrophy , disease , sense (electronics) , bioinformatics , neuroscience , genetics , gene , biology , locked nucleic acid , electrical engineering , engineering
Unlike conventional medicines, antisense oligonucleotides (ASO) work by addressing the genetic changes underlying a disease. One ASO, nusinersen, is already on the market for the treatment of spinal muscular atrophy, and several more ASO therapies in development hold potential for other neurological and neuromuscular conditions, such as Duchenne muscular dystrophy.