Intracellular expression and function of antisense catalytic RNAs

D Castanotto, M Scherr, JJ Rossi - Methods in enzymology, 2000 - Elsevier
Publisher Summary Ribozymes represent a relatively new approach for specific inactivation
of gene expression. To date, ribozymes have been reported to be effective in cell culture
against a variety of RNA targets, including viral, endogenous, and transfected sequences.
Ribozymes have been utilized to functionally destroy mRNAs as well as for the repair of
mutant RNAs. These RNA catalysts have progressed to the stage where both hammerhead
and hairpin ribozymes are currently being tested in human clinical trials. The effective use of …