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Transfection by cationic gemini lipids and surfactants

机译:阳离子双子星脂质和表面活性剂的转染

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摘要

Diseases that are linked to defective genes or mutations can in principle be cured by gene therapy, in which damaged or absent genes are either repaired or replaced by new DNA in the nucleus of the cell. Related to this, disorders associated with elevated protein expression levels can be treated by RNA interference via the delivery of siRNA to the cytoplasm of cells. Polynucleotides can be brought into cells by viruses, but this is not without risk for the patient. Alternatively, DNA and RNA can be delivered by transfection, i.e. by non-viral vector systems such as cationic surfactants, which are also referred to as cationic lipids. In this review, recent progress on cationic lipids as transfection vectors will be discussed, with special emphasis on geminis, surfactants with 2 head groups and 2 tails connected by a spacer.
机译:原则上,与缺陷基因或突变有关的疾病可以通过基因疗法治愈,其中受损或缺失的基因可以通过细胞核中的新DNA修复或替换。与此相关,与蛋白质表达水平升高相关的疾病可以通过将siRNA递送至细胞质而受到RNA干扰来治疗。多核苷酸可以被病毒带入细胞,但这对患者而言并非没有风险。或者,可以通过转染,即通过非病毒载体系统,例如阳离子表面活性剂,也称为阳离子脂质,来传递DNA和RNA。在这篇综述中,将讨论阳离子脂质作为转染载体的最新进展,特别强调双子星,具有2个头基和2个尾部(通过间隔子连接)的表面活性剂。

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