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

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