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新型基因递送系统。

Novel gene delivery systems.

作者信息

Manjila Steffy B, Baby Jomon N, Bijin Elambilan N, Constantine Icey, Pramod Kannissery, Valsalakumari Janardhanan

机构信息

College of Pharmaceutical Sciences, Government Medical College, Thiruvananthapuram, Kerala, India.

出版信息

Int J Pharm Investig. 2013 Jan;3(1):1-7. doi: 10.4103/2230-973X.108958.

Abstract

Gene therapy is an emerging field in medical and pharmaceutical sciences because of its potential in treating chronic diseases like cancer, viral infections, myocardial infarctions, and genetic disorders. Application of gene therapy is limited because of lack of suitable methods for proper introduction of genes into cells and therefore, this is an area of interest for most of the researchers. To achieve successful gene therapy, development of proper gene delivery systems could be one of the most important factors. Several nonviral and viral gene transfer methods have been developed. Even though the viral agents have a high transferring efficiency, they are difficult to handle due to their toxicity. To overcome the safety problems of the viral counterpart, several nonviral in vitro and in vivo gene delivery systems are developed. Out of these, the most promising and latest systems include polymer-based nonviral gene carriers, dendrimers, and physical means like electroporation, microinjection, etc., Shunning of possible immunogenicity and toxicity, and the feasibility of repeated administration are some of the merits of nonviral gene delivery systems over viral gene delivery. An ideal nonviral gene carrying system should possess all these merits without any compromise to its gene transferring efficiency. The viral gene delivery systems include lytic and nonlytic vectors for drug delivery. Inspite of its toxicity they are still preferred because of their long term expression, stability, and integrity. This review explores the recent developments and relevancy of the novel gene delivery systems in gene therapy.

摘要

基因治疗是医学和制药科学中的一个新兴领域,因为它在治疗癌症、病毒感染、心肌梗死和遗传疾病等慢性疾病方面具有潜力。由于缺乏将基因正确导入细胞的合适方法,基因治疗的应用受到限制,因此,这是大多数研究人员感兴趣的领域。为了实现成功的基因治疗,开发合适的基因传递系统可能是最重要的因素之一。已经开发了几种非病毒和病毒基因转移方法。尽管病毒载体具有较高的转移效率,但由于其毒性,它们难以处理。为了克服病毒载体的安全问题,人们开发了几种非病毒的体外和体内基因传递系统。其中,最有前途和最新的系统包括基于聚合物的非病毒基因载体、树枝状大分子以及电穿孔、显微注射等物理方法。避免可能的免疫原性和毒性以及重复给药的可行性是非病毒基因传递系统相对于病毒基因传递的一些优点。理想的非病毒基因携带系统应该具备所有这些优点,而不影响其基因转移效率。病毒基因传递系统包括用于药物递送的裂解和非裂解载体。尽管它们有毒性,但由于其长期表达、稳定性和完整性,仍然受到青睐。本文综述了新型基因传递系统在基因治疗中的最新进展和相关性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ff0a/3687232/1c0f3c4501cd/IJPI-3-1-g001.jpg

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