RNA干擾技術在人類疾病治療的應用進展

Applications and Progress of the Technology of RNA Interference in Human Diseases

陳秋帆(Chiu-Fan Chen)

指導教授 ： 劉炳嵐

朝陽科技大學/理工學院/應用化學系/碩士(2009年)

摘要

摘要
RNA干擾(RNA interference, RNAi)現象最初是發現存在於植物中，後來科學家發現此現象亦廣泛存在於真核生物細胞，RNAi不僅可以應用於治療疾病上，且對生物體內的生長發育和基因表達調控都有著重要的作用。在此研究中，將綜述RNAi技術在人類疾病治療上之研究與應用概況，可分為四大部份：(一)癌症治療，可透過：(1)抑制癌基因、(2)抑制突變抑癌基因、(3)干擾抗凋亡因子、(4)抑制過量表達之細胞生長因子及受體、(5)抑制病毒癌基因等方法；(二)病毒感染性疾病治療，透過：(1)抑制病毒侵入細胞、(2)抑制病毒基因或蛋白基因的表達；(三)自體免疫疾病治療，透過：(1)抑制干擾素活化、(2)抑制疾病相關之血管基因；(四)代謝性疾病治療，透過：(1)抑制代謝途徑相關基因、(2)抑制疾病蛋白過量。在這些範疇中，RNAi技術皆極具治療之潛能。
RNAi技術具有高效性、專一性地阻斷基因表達之功效，可達到基因降解(Gene knockdown)的目的。目前已有許多研究人員積極地投入RNAi研究，盡可能地了解RNAi運用機制。但RNAi技術目前尚存在著許多問題，如:如何設計高專一性siRNA、RNAi片段的運送、RNAi片段的穩定性、缺乏臨床試驗等，若能克服，相信未來RNAi技術將提供疾病治療一個新的方向。

關鍵字

代謝性疾病 ； 自體免疫性疾病 ； 病毒性疾病 ； 癌症治療 ； RNA干擾技術

並列摘要

RNA interference (RNAi) is first found in plants and scientists also found RNAi exits generally in most Eukaryotic cells. RNAi not only apply to disease therapy but also controls the growth and development as well as gene regulation. In this study, the applications and progress of RNAi technology in treating humans’ diseases were summarized. It can be divided into four parts : (A) the treatment of cancer through the inhibition of (1) cancer gene, (2) tumor suppressor gene, (3) anti-apoptosis factors, (4) overexpression of cell growth factor and its receptor, and (5) virus oncogene; (B) the treatment of the virus-infected diseases through the inhibition of (1) the attack of virus against cells, and (2) the expression of virus gene; (C) the treatment of autoimmune diseases through the inhibition of (1) the interferon activation, and (2) the blood vessel genes of the diseases; (D) the treatment of metabolic disorders by inhibiting the metabolic pathway-related genes, and overexpressive proteins of diseases. In these parts, RNAi technology is very therapeutic potential.
RNAi technology has high efficiency, specificity to block the gene expression, and achieves the purpose of gene degradation (Gene knockdown). Many researchers are currently actively engaged in RNAi research, and learn the use of RNAi mechanism as much as possible. However, RNAi technology faces many problems, such as: how to design a high specificity of siRNA, the delivery of RNAi fragment, the stability of RNAi fragment, and the lack of clinical trials. If these problems are overcome, RNAi technology will provide a new direction for the treatment of disease in the future.

並列關鍵字

metabolic diseases ； cancer therapy ； virus diseases ； autoimmune diseases ； RNA interference

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