Synthetic Hammerhead Ribozymes as Therapeutic Tools to Control Disease Genes

Title: Synthetic Hammerhead Ribozymes as Therapeutic Tools to Control Disease Genes

Volume: 5 Issue: 1

Author(s): L. Citti and G. Rainaldi

Affiliation:

Keywords: hammerhead ribozymes, gene knockdown, gene therapy, gene drugs, synthetic oligonucleotides

Abstract: Ribozymes are RNA molecules that have the ability to catalyse the cleavage and formation of covalent bonds in RNA strands at specific sites. The “hammerhead” motif, approximately 30-nucleotide long, is the smallest endonucleolytic cis-acting ribozyme structure found in natural circular RNAs of some plant viroids. Hammerhead ribozymes became appealing when it was shown that it is possible to produce trans-acting ribozymes directed against RNA sequences of interest. Since then, gene-tailored ribozymes have been designed, produced and given to cells to knock down the expression of specific genes. At present, this technology has advanced so much that many hammerhead ribozymes are being used in clinical trials. With this work we would provide some guidelines to design efficient trans-acting hammerhead ribozymes as well as review the recent results obtained with them as gene therapy tools.

Cite this article as:

Citti L. and Rainaldi G., Synthetic Hammerhead Ribozymes as Therapeutic Tools to Control Disease Genes, Current Gene Therapy 2005; 5 (1) . https://dx.doi.org/10.2174/1566523052997541