A brief history, status, and perspective of modified oligonucleotides for chemotherapeutic applications.
The advent of rapid and efficient methods of oligonucleotide synthesis has allowed the design of modified oligonucleotides that are complementary to specific nucleotide sequences in mRNA targets. Such modified oligonucleotides can be used to disrupt the flow of genetic information from transcribed mRNAs to proteins. This antisense strategy has been used to develop therapeutic oligonucleotides against cancer and various infectious diseases in humans. This overview reports recent advances in the application of oligonucleotides as drug candidates, describes the relationship between oligonucleotide modifications and their therapeutic profiles, and provides general guidelines for enhancing oligonucleotide drug properties.
Cook
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PD
Isis Pharmaceuticals, Carlsbad, California, USA.
D020029:Base Pairing; D003851:Deoxyribonucleases / Q000378:metabolism; D015195:Drug Design; D015870:Gene Expression; D049673:History, 20th Century; D009841:Oligonucleotides / Q000737:chemistry / Q000266:history / Q000627:therapeutic use*
D009841:Oligonucleotides; D003851:Deoxyribonucleases