Engineered Ribozymes: Efficient Tools for Molecular Gene Therapy and Gene Discovery

Abstract

Ribozymes are catalytic RNA molecules that cleave RNAs with high specificity. Hammerhead ribozymes are small and particularly versatile catalytic RNA molecules that cleave RNAs at specific sites (Figure 17.1A, left). The rapidly developing field of RNA catalysis is of particular current interest not only because of the intrinsic catalytic properties of ribozymes but also because of the potential utility of ribozymes as therapeutic agents and specific regulators of gene expression [16, 17, 45, 88, 89, 90, 105]. However, despite extensive efforts, the efficiency of ribozyme in vivo has generally been too low to achieve the desired biological effects. Unlike in vitro, conditions in vivo are very complex and many parameters must be taken into account, in particular conditions, the interactions of a ribozyme or its gene with intracellular proteins, which seem to be significant. Many modifications of and improvements in ribozymes, as well as methods for the introduction of ribozymes into cells, have been developed in attempts to exploit ribozymes in vivo.