Recent patents in cationic lipid carriers for delivery of nucleic acids.

Rumiana Koynova, Boris Tenchov
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引用次数: 34

Abstract

Gene therapy is a medical technique intended for treatment of disorders caused by defective, missing, or overexpressing genes. Efficient delivery vectors are necessary in order to transport genetic material to the target cells. Such vectors include viral and non-viral carriers. Viral vectors transfect cells efficiently, however risks associated with their use have limited their clinical applications. Nonviral delivery systems are safer, easier to prepare, more versatile and cost effective. However, their transfection efficiency still falls behind that of the viral vectors. Considerable research into nonviral gene delivery has been conducted in the last two decades on synthetic soft materials such as cationic lipids, polymers, surfactants, and dendrimers as prospective nucleotide carriers for gene delivery. So far, cationic lipids are the most widely used constituents of nonviral gene carriers, with multiple strategies employed to improve their in vitro and in vivo transfection. Efforts in synthesizing new cationic lipids were not fully successful in closing the gap between the efficiency of the viral vectors and that of binary cationic lipid/DNA complexes. Current efforts for improving lipofection efficiency are focused on the development of multicomponent carriers including cationic lipids as key constituents. This review summarizes the recent patents on new cationic lipids as well as on multicomponent formulations enhancing their efficiency as nucleotide carriers.

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核酸输送用阳离子脂质载体的最新专利。
基因治疗是一种用于治疗由缺陷、缺失或过度表达基因引起的疾病的医学技术。高效的传递载体是将遗传物质运送到靶细胞的必要条件。这些载体包括病毒和非病毒载体。病毒载体能有效地转染细胞,但其相关的风险限制了其临床应用。非病毒给药系统更安全、更容易制备、更通用和更具成本效益。然而,它们的转染效率仍然落后于病毒载体。在过去的二十年里,人们对非病毒基因传递进行了大量的研究,合成的软材料,如阳离子脂质、聚合物、表面活性剂和树状大分子,作为基因传递的核苷酸载体。到目前为止,阳离子脂质是非病毒基因载体中应用最广泛的成分,有多种策略可以改善它们在体外和体内的转染。合成新的阳离子脂质的努力并没有完全成功地缩小病毒载体和二元阳离子脂质/DNA复合物之间的效率差距。目前提高吸脂效率的努力主要集中在以阳离子脂质为主要成分的多组分载体的开发上。本文综述了近年来新型阳离子脂质专利以及提高其作为核苷酸载体效率的多组分制剂。
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