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Most human diseases are caused by production of abnormal proteins or malfunctioning proteins. Antisense therapy involves inhibiting production of these proteins. When a gene is known to cause a specific disease and the genetic sequence of that gene is known, it is theoretically possible to synthesize a complementary molecule that will bind to that gene and inactivate it. If you prevent the abnormal protein from being made, you prevent its damaging effects on cells, and therefore reduce or delay symptoms.
The first step in the production of a protein is the unwinding of the DNA double helix and the production of a single stranded messenger RNA molecule. The sequence of nucleotides in the mRNA molecule is called the “sense” sequence. The “antisense” molecule is the complementary RNA strand that targets the mRNA molecule and is designed to essentially “shoot the messenger” so the protein won’t be made. This therapy is also referred to as antisense oligonucleotide (ASO) therapy and as a type of gene silencing.
In January 2013, the FDA approved the antisense product Kynamro (mipomersen) to treat homozygous familial hypercholesterolemia. Vitravene (formivirsen) was approved in 1998 to treat cytomegalovirus (CMV) retinitis in AIDS patients.