Abstract
The development of gene-inactivation systems is an active and important field for both functional genomics and gene therapy. Towards this end, ribozymes (i.e. RNA enzymes), that specifically recognize and subsequently catalyze the cleavage of other target RNA molecules, are attractive molecular tools. Ribozymes represent an interesting alternative to the RNA interference (RNAi) approach for gene inactivation, especially given the fact that RNAi seems to trigger an immunological response and has demonstrated off-target effects. However, the design and optimization of a ribozymebased gene-inactivation system is not a straightforward procedure. Several aspects need to be considered in the experimental design in order to provide a suitable suppression system. In this review we present the advances in this domain made available from work using the hepatitis delta virus (HDV) ribozyme as a cis-acting RNA motif in molecular biology, as well as a trans-acting molecular scissor for the development of a gene-inactivation system. This HDV ribozyme technology possesses several unique features that are all related to the fact that it is the only catalytic cleaving RNA motif that has been discovered in humans.
Keywords: HDV ribozyme, RNA interference, antisense, ribozyme, deoxyribozyme, RNase P, functional genomic, gene therapy
Current Gene Therapy
Title: Development of Ribozyme-Based Gene-Inactivations; The Example of the Hepatitis Delta Virus Ribozyme
Volume: 7 Issue: 3
Author(s): M. Asif-Ullah, Michel Levesque, Gilles Robichaud and Jean-Pierre Perreault
Affiliation:
Keywords: HDV ribozyme, RNA interference, antisense, ribozyme, deoxyribozyme, RNase P, functional genomic, gene therapy
Abstract: The development of gene-inactivation systems is an active and important field for both functional genomics and gene therapy. Towards this end, ribozymes (i.e. RNA enzymes), that specifically recognize and subsequently catalyze the cleavage of other target RNA molecules, are attractive molecular tools. Ribozymes represent an interesting alternative to the RNA interference (RNAi) approach for gene inactivation, especially given the fact that RNAi seems to trigger an immunological response and has demonstrated off-target effects. However, the design and optimization of a ribozymebased gene-inactivation system is not a straightforward procedure. Several aspects need to be considered in the experimental design in order to provide a suitable suppression system. In this review we present the advances in this domain made available from work using the hepatitis delta virus (HDV) ribozyme as a cis-acting RNA motif in molecular biology, as well as a trans-acting molecular scissor for the development of a gene-inactivation system. This HDV ribozyme technology possesses several unique features that are all related to the fact that it is the only catalytic cleaving RNA motif that has been discovered in humans.
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Asif-Ullah M., Levesque Michel, Robichaud Gilles and Perreault Jean-Pierre, Development of Ribozyme-Based Gene-Inactivations; The Example of the Hepatitis Delta Virus Ribozyme, Current Gene Therapy 2007; 7 (3) . https://dx.doi.org/10.2174/156652307780859008
DOI https://dx.doi.org/10.2174/156652307780859008 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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