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Antisense therapy is a procedure of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is probable to generate a strand of nucleic acid (DNA, RNA or a chemical analogue) which is bind to the messenger RNA (mRNA) generated by that gene and inactivate it, efficiently turning that gene into inactive off. This is the reason of mRNA has to be single stranded for it to be translated. Otherwise, the strand might be targeted to bind a splicing site on pre-mRNA and modify the exon content of an mRNA.
This generated nucleic acid is known as an "anti-sense" oligonucleotide because its base sequence is complementary to the gene's messenger RNA (mRNA), which is known as "sense" sequence (so that a sense segment of mRNA " 5'-AAGGUC-3' " would be blocked by the anti-sense mRNA segment " 3'-UUCCAG-5' ").
Antisense drugs are being investigated to treat a variety of diseases such as cancers (including lung cancer, colorectal carcinoma, pancreatic carcinoma, malignant glioma and malignant melanoma), diabetes, Amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy and diseases such as asthma, arthritis and pouchitis with an inflammatory component. As of 2014 two antisense drugs have been approved by the U.S. Food and Drug Administration (FDA), fomivirsen (marketed as Vitravene) as a treatment for cytomegalovirus retinitis and mipomersen (marketed as Kynamro) for homozygous familial hypercholesterolemia.
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This page was last updated on December 23, 2024
