Non-viral gene therapy: Gene design and delivery
Non-viral gene therapy: Gene design and delivery
复制标题
非病毒基因治疗：基因设计和传递
DOI：
10.1007/4-431-27879-6
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发表时间：
2005
影响因子：
9.5
通讯作者：
T. Niidome
中科院分区：
文献类型：
--
作者：
K. Taira;K. Kataoka;T. Niidome
作为一种治疗严重疾病的新方法，基因作为治疗剂的潜在用途引起了人们的关注。在遗传性疾病的情况下，引入受影响基因的正常副本可能是有效的，如众所周知的由于腺苷脱氨酶(ADA)缺乏而导致严重联合免疫缺陷的基因治疗，在这种情况下，ADA的正常基因用于治疗受影响的患者。对于癌症和传染病等获得性疾病的治疗，有效的潜在策略不仅包括引入治疗性基因，如细胞因子或抗原的基因，还包括沉默异常基因的表达，其表达在疾病部分的组织中得到增强。继20世纪80年代初Sidney Altman和Thomas Cech革命性地发现核酶之后，RNA被认为是一类潜在的治疗性核酸，可能能够在临床环境中调节病毒和内源性基因的表达。2000年，我们意识到促进参与潜在治疗性基因研究的科学家和参与药物和基因递送系统研究的科学家之间互动的重要性。出于这个原因，我们帮助建立了日本基因设计和交付协会(http://www.基因传递。Org/)。同年，一种新的、强大的被称为RNA干扰(RNAi)的现象被报道。这种细胞内现象已经在包括并在进化规模上高于裂殖酵母的有机体细胞中被发现。如果能开发出合适的递送系统，RNAi的潜在抑制作用可能会比核酶大得多，从而使核酸药物的梦想更近一步。
The potential use of genes as therapeutic agents has attracted attention as a novel approach to the treatment of severe diseases. In the case of inherited disorders, the introduction of a normal copy of the affected gene can be effective, as in the wellknown case of gene therapy for severe combined immune deficiency due to adenosine deaminase (ADA) deficiency, in which the normal gene for ADA is used to treat the affected patient. For the treatment of acquired disorders, such as cancer and infectious diseases, effective potential strategies involve not only the introduction of a therapeutic gene, such as the gene for a cytokine or an antigen, but also the silencing of the expression of an abnormal gene, whose expression is enhanced in the tissue of the diseased part.Subsequent to the revolutionary discovery of ribozymes by Sidney Altman and Thomas Cech in the early 1980s, RNAs were recognized as a class of potentially therapeutic nucleic acids that might be able to regulate the expression of viral and endogenous genes in a clinical setting. In 2000, we realized the importance of stimulating interactions between scientists who are involved in research on potentially therapeutic genes and those who are involved in studies of drug-and gene-delivery systems. For this reason, we helped to establish the Japanese Society of Gene Design and Delivery (http://www. gene-delivery. org/) in 2001. That same year, the new and powerful phenomenon known as RNA interference (RNAi) was reported. This intracellular phenomenon has been recognized in cells of organisms that include and are higher on the evolutionary scale than Schizosaccharomyces pombe. The potential suppressive effects of RNAi might be expected to be significantly greater than those of ribozymes, bringing the dream of nucleic acid drugs closer, provided that suitable delivery systems can be developed.