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首页> 外文期刊>Expert opinion on biological therapy >Non-viral gene delivery: from the needle to the nucleus.
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Non-viral gene delivery: from the needle to the nucleus.

机译：非病毒基因传递：从针头到细胞核。

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摘要

Despite nearly two decades of research, the successful application of systemically delivered non-viral gene therapies to treat human disease is still limited by poor transfection efficiency. The major barriers in the circulation and in the cell that limit transfection efficiency have been identified and the field has entered a phase of design and testing of more sophisticated carrier systems that attempt to circumvent these barriers. These studies are increasingly conducted in vivo using rapid quantitative measures of gene transfer efficiency as a guide. Although there has been steady progress in developing DNA nanoparticles that navigate the circulation, enter the target cell and escape lysosomal targeting, the final goal of efficiently traversing the nuclear membrane remains the most significant challenge. The ultimate goal is to develop elegant delivery systems that work in concert to deliver DNA from the needle to the nucleus.
机译：尽管进行了将近二十年的研究，但转染效率差仍然限制了系统递送的非病毒基因疗法在治疗人类疾病中的成功应用。已经确定了限制转染效率的循环和细胞中的主要障碍，并且该领域已进入设计和测试更复杂的载体系统的阶段，这些系统试图绕过这些障碍。这些研究越来越多地使用基因转移效率的快速定​​量方法作为指导，在体内进行。尽管开发可导航循环，进入靶细胞并逃避溶酶体靶向的DNA纳米颗粒已取得稳步进展，但有效穿越核膜的最终目标仍然是最重大的挑战。最终目标是开发优雅的输送系统，这些系统可以协同工作，将DNA从针头输送到细胞核。

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