Applications of nanoparticle systems in gene delivery and gene therapy

Abstract

For successful gene therapy, expansion of appropriate gene delivery systems could be one of the factors of major significance. Gene therapy provides large opportunities for treating diseases, including genetic disorders, infections, and cancer. Polymeric carriers have relatively low cytotoxicity and immunogenicity. Polymeric gene carriers are a potential substitute to using viral vectors. Overall, polymeric carriers can contain large-sized DNA, be conjugated with suitable functionalities, and be administered frequently. However, polymeric gene carriers have some restrictions, such as low gene transfection efficiencies and a moderately short period of gene expression.

This study explores the current status of development of polymeric gene carriers, and presents guidelines for the prospective use of the polymer-based gene delivery systems in gene therapy.

Keywords::

• gene delivery
• gene therapy
• gene carriers
• immunogenicity

Authors’ contributions

AA and TK conceived of the study and participated in its design and coordination. EA participated in the sequence alignment and drafted the manuscript. All authors read and approved the final manuscript.

Acknowledgments

The authors thank the Department of Medical Nanotechnology, Faculty of Advanced Medical Science, Tabriz University, for all support provided. This work is funded by the Grant 2011-0014246 of the National Research Foundation of Korea. This work is also funded by the 2014 Drug Applied Research Center Tabriz University of Medical Sciences Grant.

Declaration of interest

The authors report no declarations of interest. The authors alone are responsible for the content and writing of the paper.