Recent developments and perspectives on gene therapy using synthetic vectors

Abstract

Nonviral vector technology is attracting increasing importance in the biomedical community owing to unique advantages and prospects for the treatment of severe diseases by gene therapy. In this review, synthetic vectors that allow the controlled design of efficient and biocompatible carriers are highlighted. The current benefits, potentials, problems and unmet needs of synthetic gene delivery systems, as well as the strategies to overcome the obstacles are also discussed. Common design principles and structure-activity trends have been established that are important for stable and targeted transport to regions of interest in the body, efficient uptake into cells as well as controlled release of drugs inside the cells, for example, in specialized compartments. The status quo of the use of these systems in preclinical and clinical trials is also considered.