Antisense therapeutic agents design

In order to obtain therapeutic agents based on synthetic ribozymes, it is necessary to modify these structures chemically. Such modifications - as in the traditional antisense strategy - should confer resistance to nucleases, selectivity, and proper hybridization and uptake characteristics. In the case of ribozymes, the design of new modified nucleotides becomes more complex, since correct folding of the nucleic acid is needed in order to maintain the catalytic activity. Several studies (e.g.. X-ray structure elucidation and mapping with modified nucleotides) have shown that the presence of the 2 -hydroxyl group at specific positions in the catalytic core is essential for hydrolytic activity. Ribozymes are currently used in larger screens as a... 

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