DNA载体拓扑结构对非病毒基因治疗的安全性和有效性的影响

Title:Impact of DNA Vector Topology on Non-Viral Gene Therapeutic Safety and Efficacy

Volume: 14 Issue: 4

Author(s): Chi H. Sum, Shawn Wettig and Roderick A. Slavcev

Affiliation:

关键词: 阳离子脂质体，基因治疗，线性共价闭合（LCC）DNA 迷你载体，核酸复合物，非病毒基因载体，重组质粒DNA载体，合成载体

摘要: Gene therapy continues to grow as an emerging treatment strategy toward numerous diseases. However, such prospects are hindered by the use of viral vectors prompting significant safety concerns along with limitations concerning repeat administrations, size of delivered gene construct, scale-up, high production costs, contamination during production, and lack of desired tissue selectivity. Non-viral gene delivery demonstrates the potential to address the abovementioned limitations, but itself generally suffers from low efficacy. Continuing efforts have been made to develop innovative delivery systems, synthetic gene carriers, and DNA vectors in a concerted attempt to enhance gene delivery suitable for clinical applications. In this review, we focus on the advances in the design of novel DNA vectors catered to enhance transfection and transgene expression and their influences on the efficacy and safety of existing and emerging delivery systems and synthetic vectors for non viral gene delivery.

Cite this article as:

Sum H. Chi, Wettig Shawn and Slavcev A. Roderick, Impact of DNA Vector Topology on Non-Viral Gene Therapeutic Safety and Efficacy, Current Gene Therapy 2014; 14 (4) . https://dx.doi.org/10.2174/1566523214666140612154929