STERICALLY STABILIZED CATIONIC LIPID - DNA COMPLEXES USED IN GENE DELIVERY

这个子项目是利用资源的许多研究子项目之一。 由NIH/NCRR资助的中心拨款提供。对子项目的主要支持 子项目的首席调查员可能是由其他来源提供的， 包括美国国立卫生研究院的其他来源。为子项目列出的总成本可能 表示该子项目使用的中心基础设施的估计数量， 不是由NCRR赠款提供给次级项目或次级项目工作人员的直接资金。 用于治疗的无病毒合成载体的研究领域 尤其是使用基于阳离子脂质体的载体的应用最近经历了 文艺复兴。这是由于载体的非免疫原性和潜在的 转移较大的DNA片段，如约1 Mbps的人类人造DNA片段 将染色体导入细胞，这在病毒载体上是不可行的。然而，转染法 效率(TE；一种外源基因表达的度量，该基因被转移到 细胞)的阳离子脂质-DNA复合体(CDC)，与病毒载体和 只有我们对发展中国家和发展中国家之间的相互作用的了解有了显著的进一步增加 细胞将在体外以及体内对TE进行优化。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. The research field focusing on virus-free synthetic vectors intended for therapeutic delivery applications, in particular, using cationic liposome based vectors has recently undergone a renaissance. This is due to the non-immunogenicity of the vectors and the potential of transferring large DNA pieces such as sections of about 1Mbps of human artificial chromosome into cells which is not feasible with viral vectors. However, transfection efficiency (TE; a measure of the expression of an exogenous gene that is transferred into cells) of cationic lipid-DNA complexes (CLDCs), remains low compared to viral vectors and only a significant further increase in our knowledge of the interactions between CLDCs and cells will result in in vitro as well as in vivo optimization of TE.