摘要
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 transfers the health genes into cells to replace the disease-causing genes by utilization of a viral or non-viral carrier. The human diseases of at least 1200 have been reported to be caused by defective genes. One of the most reasonable choices to treat these diseases is gene therapy. The key barrier to overcome in gene therapy is to resolve the issue： which kind of carriers can transport the health genes into the target cells safely and efficiently. The non-viral gene carriers, such as organic cations and cationic polymers, have attracted intensive attention. However, metal complexes, nanoparticles and calcium phosphate have also been recognized as a promising nucleic acid-delivering carrier in recent years. In this paper, the studies on the nucleic acid-delivering functions of metal complexes are reviewed. We expect to be capable of understanding advantages and disadvantages of metal complexes as a promising gene carrier.
出处
《中国科学：化学》
CAS
CSCD
北大核心
2014年第4期495-505,共11页
SCIENTIA SINICA Chimica
基金
国家自然科学基金(20971049
21072074
21271079)资助
