Gene silencing
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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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