Synthetic and natural polycations for gene therapy: state of the art and new perspectives.
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.
Tiera
Marcio J
MJ
Orthopedic Research Laboratory, Hôpital du Sacré-Caeur de Montréal, Université de Montréal, Montreal, QC Canada H4J 1C5.
Winnik
Fran Oise M
FO
Fernandes
Julio C
JC
D000818:Animals; D002412:Cations / Q000008:administration & dosage* / Q000737:chemistry; D016503:Drug Delivery Systems; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D006801:Humans; D049329:Nanostructures; D011108:Polymers / Q000008:administration & dosage* / Q000737:chemistry
D002412:Cations; D011108:Polymers