工業技術研究院

技術簡介

開發新型的非病毒基因載體，可有效的遞送各類基因藥物包括Anti-sense ODN、plasmid DNA及siRNA、miRNA等基因治療物質至生體內。此基因載體的原理是利用具細胞標的特性的ligands結合lipid-based的配方設計，可針對特定性質的疾病細胞或組織做標靶治療。此非病毒基因載體系統不僅具有專一性、低毒性且具有血清安定性，可使基因藥物在生體內的遞送效率及功效大幅提高。此技術與一般載體最大的不同點在於，能夠同時標的至細胞膜與細胞核(Double Targeting)，且具有pH sensitive 的特點，可以在細胞內programmable的釋放基因物質使其進入細胞核中。這些特徵可使本技術成為研究方面以及臨床應用的有利工具。

Abstract

The novel non-viral gene carrier developed can effectively deliver genetic materials, including anti-sense ODN, plasmid DNA, siRNA and miRNA to target tissue in vivo. The delivery concept of this gene carrier is by taking the advantage of ligand-receptor binding affinity combined with special lipid-based formulation design to promote the delivery of genetic materials to specific cell type for targeted gene therapy. In addition to the active targeting ability, the non-viral gene carrier has very low cytotoxicity and high serum stability. This novel delivery system not only has high specificity, low toxicity and serum stability, but also greatly improves the delivery efficiency of genetic materials and their efficacy. The most distinguished feature of this novel gene carrier is its ability to target to both cell membrane and nucleus (Double Targeting). Moreover, the gene carrier itself is pH sensitive, and the release of contained genetic materials is programmable and well-controlled for different purposes. All these intelligent features make this novel gene carrier a powerful tool for in vivo delivery and therapeutics.

技術規格

包覆率 > 95% 粒徑大小 < 100nm 電中性 血清安定性 > 48小時 pH值敏感性釋放 主動標的傳輸

Technical Specification

Encapsulation Efficiency (%) > 95% Particle Size < 100nm Neutral Serum Stability > 48h pH sensitive release Active Targeting delivery

技術特色

將基因藥物穩定的在生體中遞送到目標組織及細胞，發揮調控基因的功能，並達到治療的效果。此技術目前已具備包含製程建立、關鍵新材料開發、規格設定、功效評估、前臨床試驗與臨床試驗之規畫等，屬完整且系統化的基因載體技術開發。

應用範圍

此基因藥物遞送技術可應用在基因體、蛋白質體研究、疾病機制探討以及臨床基因治療等方面

接受技術者具備基礎建議（設備）

細胞培養相關設備 動物疾病評估模型 化合物合成相關設備

接受技術者具備基礎建議（專業）

臨床試驗相關知識或經驗 藥物開發相關經驗 試劑開發相關經驗

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