404
Antisense Oligonucleotides as Potential Drugs
9
Conclusion
413
Bibliography
413
Books and Reviews
413
Primary Literature
414
Keywords
Anneal (hybridize)
To form doubled-stranded oligo/polynucleotides (duplex) from two complementary
single-stranded polynucleotides by heating and cooling.
Antisense
A strand of DNA that has the same sequence as mRNA (coding strand).
Ribozyme
Nucleic acid polymer (RNA) that catalyzes a chemical reaction, mostly
transesteriFcation.
RNase H
A ribonuclease that recognizes and cleaves internally the RNA strand of a
DNA–RNA hybrid.
RNAi
RNA interference, a natural process whereby small double-stranded (21-mers) RNAs
induce gene silencing.
Phosphorothioate
Oligonucleotides in which one of the nonbinding oxygens on phosphorus is replaced
by sulfur.
Backbone
Refers to the sugar–phosphate linkage in DNA or RNA that can be replaced by
isosteric linkages.
¥
Antisense technology is a novel method for drug discovery and drug development.
Antisense oligodeoxynucleotides (ODN) work at the genetic level by inhibiting the
translation of disease-causing proteins.
Proteins are the key players in the etiology of diseases. Especially for cancer and
viral infections, the antisense technology shows promise. The underlying mechanism
of this approach is to sequence-speciFcally interfere in the genetic flow at the mRNA
level. This molecular recognition is believed to rely on Watson–Crick base pairing
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