Gene Transfer Using Nonviral Delivery Systems
Gene Transfer Using Nonviral Delivery Systems
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DOI：
10.1177/089686080602600603
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发表时间：
2006-11
影响因子：
2.8
通讯作者：
M. Miyazaki;Y. Obata;K. Abe;A. Furusu;T. Koji;Y. Tabata;S. Kohno
中科院分区：
文献类型：
--
作者：
M. Miyazaki;Y. Obata;K. Abe;A. Furusu;T. Koji;Y. Tabata;S. Kohno
在腹膜透析中，腹膜功能丧失是治疗失败的主要因素。腹膜功能的改变与腹膜的结构变化有关，包括腹膜硬化和细胞外基质增加。虽然腹膜硬化被认为在某种程度上是可逆的，通过腹膜休息，这改善了腹膜功能，促进形态学变化，一直没有治疗干预，也没有药物对腹膜硬化的发展和进展。利用基因工程的最新生物技术进展，基于腹膜遗传修饰的策略可能是一种潜在的治疗腹膜硬化和腹膜衰竭的策略。在这种基因治疗可能应用于临床之前，必须建立安全有效的基因递送系统以及基因治疗方法的选择。目前有两种类型的基因转移载体：病毒和非病毒。病毒载体在持续性非卧床腹膜透析研究领域主要用作基因递送系统，然而，它们存在诸如免疫原性和毒性的若干问题。另一方面，非病毒载体比病毒载体具有几个优点。我们在这里审查基因转移使用非病毒载体系统在腹膜：电穿孔，脂质体和阳离子明胶微球。在腹膜透析领域，使用非病毒载体的基因治疗研究目前是有限的。递送方法的改进以及靶向基因的智能设计已经在非病毒载体的效率、特异性和时间控制方面带来了很大程度的增强。
In peritoneal dialysis, loss of peritoneal function is a major factor in treatment failure. The alterations in peritoneal function are related to structural changes in the peritoneal membrane, including peritoneal sclerosis with increased extracellular matrix. Although peritoneal sclerosis is considered reversible to some extent through peritoneal rest, which improves peritoneal function and facilitates morphological changes, there has been no therapeutic intervention and no drug against the development and progression of peritoneal sclerosis. Using recent biotechnological advances in genetic engineering, a strategy based on genetic modification of the peritoneal membrane could be a potential therapeutic maneuver against peritoneal sclerosis and peritoneal membrane failure. Before this gene therapy may be applied clinically, a safe and effective gene delivery system as well as the selection of a gene therapy method must be established. There are presently two kinds of gene transfer vectors: viral and nonviral. Viral vectors are used mainly as a gene delivery system in the field of continuous ambulatory peritoneal dialysis research; however, they have several problems such as immunogenicity and toxicity. On the other hand, nonviral vectors have several advantages over viral vectors. We review here gene transfer using nonviral vector systems in the peritoneum: electroporation, liposomes, and cationized gelatin microspheres. In the field of peritoneal dialysis, gene therapy research using nonviral vectors is presently limited. Improvement in delivery methods together with an intelligent design of targeted genes has brought about large degrees of enhancement in the efficiency, specificity, and temporal control of nonviral vectors.