ANTISENSE TECHNOLOGY
ANTISENSE TECHNOLOGY An antisense oligonucleotide (ASO) is a single-stranded deoxyribonucleotide, which is complementary to the mRNA target. The goal of the antisense approach is the downregulation of a molecular target, usually achieved by induction of RNase H endonuclease activity that cleaves the RNA-DNA heteroduplex with a significant reduction of the target gene translation . Other ASO-driven mechanisms include inhibition of 5′ cap formation, alteration of splicing process (splice-switching), and steric hindrance of ribosomal activity . Drug # There are six FDA-approved drugs based on oligonucleotide therapy:- (1) fomivirsen for treatment of CMV retinitis in AIDS patients. (2) mipomersen for treatment of familial hypercholesterolemia. (3) Defibrotide for treatment of veno-occlusive disease in the liver. (4) eteplirsen for the treatment of Duchenne muscular dystrophy. (5) pegaptanib for the treatment of neovascular age-related macular degeneration. (6) nusinersen ...