Abstract
Currently, the major drawback of gene therapy is the gene transfection rate. The two main types of vectors that are used in gene therapy are based on viral or non-viral gene delivery systems. There are several non-viral systems that can be used to transfer foreign genetic material into the human body. In order to do so, the DNA to be transferred must escape the processes that affect the disposition of macromolecules. These processes include the interaction with blood components, vascular endothelial cells and uptake by the reticuloendothelial system. Furthermore, the degradation of therapeutic DNA by serum nucleases is also a potential obstacle for functional delivery to the target cell. Cationic polymers have a great potential for DNA complexation and may be useful as non-viral vectors for gene therapy applications. The objective of this review was to address the state of the art in gene therapy using synthetic and natural polycations and the latest strategies to improve the efficiency of gene transfer into the cell.
Keywords: Polymers, nanoparticles, gene therapy, DNA, polycations
Current Gene Therapy
Title: Synthetic and Natural Polycations for Gene Therapy: State of the Art and New Perspectives
Volume: 6 Issue: 1
Author(s): Marcio J. Tiera, Francoise M. Winnik and Julio C. Fernandes
Affiliation:
Keywords: Polymers, nanoparticles, gene therapy, DNA, polycations
Abstract: Currently, the major drawback of gene therapy is the gene transfection rate. The two main types of vectors that are used in gene therapy are based on viral or non-viral gene delivery systems. There are several non-viral systems that can be used to transfer foreign genetic material into the human body. In order to do so, the DNA to be transferred must escape the processes that affect the disposition of macromolecules. These processes include the interaction with blood components, vascular endothelial cells and uptake by the reticuloendothelial system. Furthermore, the degradation of therapeutic DNA by serum nucleases is also a potential obstacle for functional delivery to the target cell. Cationic polymers have a great potential for DNA complexation and may be useful as non-viral vectors for gene therapy applications. The objective of this review was to address the state of the art in gene therapy using synthetic and natural polycations and the latest strategies to improve the efficiency of gene transfer into the cell.
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Tiera J. Marcio, Winnik M. Francoise and Fernandes C. Julio, Synthetic and Natural Polycations for Gene Therapy: State of the Art and New Perspectives, Current Gene Therapy 2006; 6 (1) . https://dx.doi.org/10.2174/156652306775515510
DOI https://dx.doi.org/10.2174/156652306775515510 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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