Limitations of Clinical Application of siRNA Delivery Based on Non-Viral Vectors

Document Type : Review Article

Authors

1 Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran

2 Assistant Professor, Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran

Abstract

In recent years, much more attentions have been focused on siRNA-based gene therapy. This approach is based on PTGS (Post-transcriptional gene silencing). Due to some defects in viral vectors, non-viral vectors have been used to deliver nucleic acids into target cells. Although, transfection efficiency in non-viral vectors is less than viral ones, but safety of non-viral vectors is much more. Characteristic features of siRNA, such as high compatibility, application in low doses and its versatility make it suitable in the gene therapy field. However, some challenges, such as stimulating immune system and Off-target silencing will be remain. In this review article, we express bottlenecks existing in siRNA delivery into target cells. According to the information, with further development of siRNA delivery in the future, it could be a promising approach in treatment for a variety of genetic diseases.

Keywords


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