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基因治疗是指利用一种载体将健康的基因载入细胞替换致病的基因.由基因缺陷导致的人类疾病达1200多种,最合理的选择是采用基因替换的方法进行治疗.基因治疗的关键问题是解决“使用何种载体才能安全有效地将治疗基因载入靶细胞”.非病毒基因载体主要是一些有机阳离子物种,一直受到极大重视;近年来,磷酸钙、纳米粒子和金属配合物释放核酸的功能也开始受到关注.本文总结了金属配合物作为非病毒基因载体使用的研究进展,希望由此理解配合物释放核酸的优势和不足之处.
Gene therapy refers to the use of a vector to load healthy genes into cells to replace disease-causing genes, and more than 1,200 human diseases caused by genetic defects are the most reasonable options for gene replacement therapy. The problem is to solve “what kind of vector is used to safely and efficiently load the therapeutic gene into the target cell.” Non-viral gene vectors are mainly organic cationic species and have received great attention; in recent years, calcium phosphates, nanoparticles and metals The function of the complex to release nucleic acid has also begun to attract attention.This article summarizes the metal complexes as a non-viral gene carrier research progress, hope to understand the advantages and disadvantages of the complex nucleic acid release.