EXPRESSION OF RIBOZYMES IN GENE-TRANSFER SYSTEMS TO MODULATE TARGET RNA LEVELS

The possibility of designing ribozymes to cleave any specific target R NA has rendered them valuable tools in both basic research and therape utic applications. In the therapeutics area, they have been exploited to target viral RNAs in infectious diseases, dominant oncogenes in can cers and specific somatic mutations in genetic disorders. Most notably , several ribozyme gene therapy protocols for HIV patients are already in Phase 1 trials. More recently, ribozymes have been used for transg enic animal research, gene target validation and pathway elucidation.