Zhang Shubiao, Xu Yingmei, Wang Bing, Qiao Weihong, Liu Dongliang, Li Zongshi
Department of Chemical Engineering, Dalian Nationalities University, Dalian Development Zone 116600, PR China.
J Control Release. 2004 Nov 24;100(2):165-80. doi: 10.1016/j.jconrel.2004.08.019.
Gene transfer represents an important advance in the treatment of both genetic and acquired diseases. Many cationic lipids and cationic polymers naturally occurred or synthesized have been used for gene transfer. They have the advantages over viral gene transfer as non-immunogenic, easy to produce and not oncogenic. These cationic compounds, however, have the major limitations of inefficient transfection and toxicity to cells. For overcoming these problems, many new cationic compounds were developed since the first cationic lipid, DOTMA, was found usage in gene therapy. This article reviews cationic lipids for gene therapy from chemistry viewpoint and we classify these compounds as monovalent cationic lipids, polyvalent cationic lipids, cationic polymers, guanidine containing compounds, cationic peptides and cholesterol containing compounds, and hope to provide suggestions on the development of this variety of cationic compounds through the discussion.
基因转移代表了在治疗遗传性疾病和后天性疾病方面的一项重要进展。许多天然存在或合成的阳离子脂质和阳离子聚合物已被用于基因转移。它们相对于病毒基因转移具有非免疫原性、易于生产且无致癌性的优势。然而，这些阳离子化合物存在转染效率低和对细胞有毒性的主要局限性。为了克服这些问题，自第一种阳离子脂质二油酰基磷脂酰乙醇胺（DOTMA）被用于基因治疗以来，许多新的阳离子化合物被开发出来。本文从化学角度综述了用于基因治疗的阳离子脂质，并将这些化合物分为单价阳离子脂质、多价阳离子脂质、阳离子聚合物、含胍化合物、阳离子肽和含胆固醇化合物，希望通过讨论为这类阳离子化合物的开发提供建议。
