Gene silencing

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Chapter: Pharmaceutical Drugs and Dosage: Biotechnology-based drugs

Antisense drugs inhibit the existing but abnormally expressed genes by blocking the transcription of DNA or the translation of mRNA.


Gene silencing

Antisense drugs inhibit the existing but abnormally expressed genes by blocking the transcription of DNA or the translation of mRNA.


Figure 26.1 Mode of action of nucleic acids. Gene therapy aims at producing therapeutic proteins, whereas antisense therapy aims at blocking the production of aberrant proteins.

Figure 26.1 illustrates the different modes of action of antisense com-pounds. Overexpression of a particular protein can lead or contribute to a disease state, such as fibrosis and cancer. Antisense drugs are used to stop the production of these aberrant proteins. Antisense drugs work at the genetic level to interrupt the process by which disease-causing proteins are produced. This is true of both host diseases (such as cancer) and infectious diseases (such as acquired immune deficiency syndrome [AIDS]).

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