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內科學誌 Scopus

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篇名 Ultrasound-based and Non-viral Technologies in Gene Therapy
卷期 18:4
並列篇名 現代超音波及非病毒性基因治療科技
作者 蘇正煌葉宏一侯嘉殷;周友三蔡正河
頁次 167-181
關鍵字 Gene therapyVectorUltrasoundCavitationMicrobubbleTransfection efficiencyScopusTSCI
出刊日期 200708

中文摘要

所謂的基因治療(gene therapy)是指利用分子生物學中DNA重組(DNA recombination)以及轉殖(transgenetic)的技術,把重組後之DNA分子傳送至體細胞內,以達成預防或治療疾病的現代醫療科技。由於病毒載體(viral vector)於生物體內不可預知的毒性及免疫反應,所以非病毒載體(non-viral vector),如聚合物(polymer),成為另一種載體選擇。超音波由於其物理特性-超音波性空洞形成(acoustic cavitation),尤其是瞬時超音波空洞現象(inertial cavitation),可增強大分子如質體DNA(plasmid DNA)對細胞膜的通透性,並在細胞膜上形成顯微小孔。超音波用微氣泡(ultrasound microbubble contrast agent)更能進一步降低超音波輸出能量,減少細胞毒性。超音波基因傳送(gene delivery)合併應用微氣泡及非病毒載體,如聚合物,可進一步增強基因轉移效能。根據上述觀察,本文描述基因傳送過程中可能經歷之障礙及可運用之載體與傳送方法,並介紹目前超音波基因傳送的細胞及動物模式,提供臨床醫師及研究工作者另一種思考。

英文摘要

Gene therapy is a technique for the purpose of correcting or preventing a disease by delivering genes into an individual's cells and tissues. Gene therapy is still in its infancy and at an experimental stage. Synthetic vectors are considered to be a prerequisite for gene deliveries, as viral vectors have fundamental problems in relation to safety issues, as well as large-scale production. Among the physical approaches, ultrasound with its bioeffects-acoustic cavitation, especially inertial cavitation,can increase the permeability of cell membrane to macromolecules such as plasmid DNA. Microbubbles, or ultrasound contrast agents, lower the threshold for cavitation by ultrasound energy. Furthermore, ultrasound-enhanced gene delivery using polymers or other non-viral vectors, though also in its preclinical stage may hold a lot of promise for the future. The aims of this brief review focus on understanding of the barriers to gene transfer and useful vectors or tools that are applied in gene delivery and on introducing the feasible models in terms of ultrasound-based gene delivery.

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