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 for the management of spinal muscular atrophy.



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