Oligonucleotide Drugs

A recent application of DNA technology has been the development of oligonucleotide drugs, which are short sequences of synthetic DNA or RNA molecules that can be used to treat diseases. Antisense oligonucleotides are complementary to undesirable RNAs, such as viral RNA. When added to a cell, these antisense DNAs bind to the viral mRNA and inhibit its translation.

Single-stranded DNA oligonucleotides bind tightly to other DNA sequences, forming a triplex DNA molecule (iFigure 18.25). The formation of triplex DNA interferes with the binding of RNA polymerase and other proteins required for transcription. Other oligonucleotides are ribozymes, RNA molecules that function as enzymes (see Chapter 13). These compounds bind to specific mRNA molecules and cleave them into fragments, destroying their ability to encode proteins. Several oligonucleotide drugs are already being tested for the treatment of AIDS and cancer.

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