Antisense oligonucleotides: A new class of potential anti-aids and anticancer drugs
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The control of gene expression using antisense oligonucleotides holds the potential to provide an important new paradigm for human therapeutics. One of the features that distinguishes oligonucleotide-based therapy from other approaches is the high degree of selectivity that is available. The most impressive results so far have been obtained with phosphorothioate oligonucleotides, despite some non-antisense and potentially toxic effects. Sequence-specific antisense effects were observed with oligomers of 11-15 nucleotide sequences. Multiple oligonucleotides are directed to the target RNA to enhance specificity and to obtain synergistic effect. Advances in nucleic acid chemistry have fueled progress in the antisense technology; nuclease-resistant oligonucleotides now come with various backbone modifications, some of which also enhance the specificity of interaction with target RNA. Clinical trials of antisense oligonucleotides in AIDS and cancer are underway in several clinical centers. The search for new classes of oligonucleotides with desirable features for various viral infections and several genetic disorders would be central future prospects in antisense research. Hopefully, successive chemical refinements with improved bioavailability and selectivity of oligonucleotides, and delivery formulations, will result in a powerful new class of anti-AIDS and anticancer drugs in the near future.
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