Antisense drug

Antisense drug refers to a form of treatment that uses antisense therapy to target specific molecular genetic mechanisms at the RNA level. This approach is used to alter gene expression and can be applied in the treatment of various diseases, including cancer, viral diseases, and genetic disorders.

History

The concept of antisense drugs was first proposed in the 1970s, following the discovery of the DNA double helix structure and the understanding of the central dogma of molecular biology. The first antisense drug, Fomivirsen, was approved by the FDA in 1998 for the treatment of cytomegalovirus retinitis.

Mechanism of Action

Antisense drugs work by binding to the mRNA produced by a specific gene. This binding can prevent the mRNA from being translated into protein, effectively silencing the gene. This mechanism of action allows antisense drugs to target and inhibit specific genes that contribute to disease.

Applications

Antisense drugs have been developed for a variety of diseases. For example, Nusinersen is an antisense drug used to treat spinal muscular atrophy, a genetic disorder that causes muscle weakness and atrophy. Other antisense drugs, such as Mipomersen, are used to treat hypercholesterolemia, a condition characterized by high levels of cholesterol in the blood.

Challenges and Future Directions

Despite the potential of antisense drugs, there are several challenges to their development and use. These include issues related to drug delivery, stability, and potential off-target effects. However, ongoing research and technological advancements continue to improve the efficacy and safety of antisense drugs.