Balkan Journal of Medical Genetics

RNA enzymes (Ribozymes)- gene therapy applications for clinical medicine
Toudjarska I1, Kremensky IM1, Mitev VI2*
*Corresponding Author: Professor Dr. Vanio I Mitev e-mail: mitev@medfac.acad.bg
page: 3

Perspectives

Since the discovery of RNA catalysts, we have come a long way in our understanding of their nature and possible applications. In the past few years, a number of clinical trials have been initiated to begin to evaluate the safety and efficacy of variety of innovative RNA-based therapeutics. RNA enzymes that can inhibit gene expression, block protein function or repair defective gene transcripts raise significant interest for gene therapy applications in clinical medicine. Moreover, the scientific community works to develop new and improved delivery vehicles, safer gene therapy vectors, and better animal models for their pre-clinical and clinical studies, to harness the utility of ribozymes to protect and improve human life. Figure 1 (a) Schematic representation of the 2D-structure of hammerhead and hairpin ribozymes. Arrows denoted the positions of the cleavage sites. (b) Schematic representation of trans-acting hammerhead ribozyme. After recognition of the target sequence and binding, target mRNA is cleaved and the products released for multiple turnover. Table 1 Naturally occurring ribozymes and ribonucleoprotein enzymes. In Table 1 are outlined the different groups of ribozymes, the type of chemical reactions they catalyze, their size and relative abundance.

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