ABSTRACT
This chapter provides an overview of the current status of ribozyme and triplex deoxyribonucleic acid (DNA) technology. It attempts to point out the therapeutic potential of the oligonucleotide-based drugs against infectious agents and degenerative disorders. The hammerhead ribozyme is one of the smallest RNA enzymes. Regulation of gene expression can occur at several levels, including transcription, mRNA stabilization, and translation. One oligonucleotide-based approach to inhibit expression of specific genes is to target the corresponding mRNA for enzymatic degradation. The cleavage of the RNA substrate by the hammerhead ribozyme occurs via a transesterification reaction which generates 5’ hydroxyl and 2’-3’ cyclic phosphate termini. The hammerhead ribozyme transcripts usually accumulate to relatively low levels and only within the cytoplasm. With improvements in intracellular stability, delivery methods, and oligonucleotide design, and with rapid advances in our knowledge of gene sequences, oligonucleotide technology is certain to become a major player in gene therapy.
