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- Wednesday, February 4, 2004
- Pharmacology and Therapeutics of Anti-sense Oligodeoxynucleotides
- Published at:Not Found
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Pharmacology and Therapeutics of Anti-sense Oligodeoxynucleotides
Waleed Sweileh
College of Pharmacy, An-Najah National University, Nablus, Palestine
E-mail: [email protected]
Received: (18/9/2002), Accepted: (9/12/2003)
Abstract
The purpose of this review is to provide pharmacists and pharmacologists with a
background information about current therapeutic potentials and clinical trials on the
anti-sense oligonucleotides (AS oligos). Anti-sense oligonucleotides are short, modified
single stranded DNA or RNA that hybridize with target mRNA and inhibit synthesis of
encoded peptide. This inhibition is achieved by either degradation of target mRNA by
RNase enzyme or by blocking translation. The specificity of hybridization makes antisense
treatment an attractive strategy to selectively modulate the expression of genes
involved in the pathogenesis of diseases. Anti-sense oligos have now reached phase I
and II in clinical trials for the treatment of cancer and viral infections. One anti-sense
drug has been approved for local treatment of retinitis induced by cytomegalovirus. A
number of important challenges for the development of anti-sense oligonucleotides in
the clinical use have been identified, including stability, cellular uptake, target
sequence selection, oligonucleotide: protein interactions, and cost of manufacture.
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