STERICALLY STABILIZED CATIONIC LIPID - DNA COMPLEXES USED IN GENE DELIVERY

空间稳定的阳离子脂质 - 用于基因传递的 DNA 复合物

• 批准号: 8362475
• 负责人: CYRUS R SAFINYA
• 金额: $ 1.93万
• 依托单位: SCRIPPS RESEARCH INSTITUTE, THE
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-05-01 至 2012-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8362475
• 关键词: Cells; Chromosomes, Artificial, Human; Complex; DNA; Funding; Gene Delivery; Genes; Grant; In Vitro; Knowledge; Lipids; Liposomes; Measures; Microscopy; Molecular; National Center for Research Resources; Principal Investigator; Renaissance; Research; Research Infrastructure; Resources; Source; Therapeutic; Transfection; United States National Institutes of Health; Viral Vector; Virus; base; cost; in vivo; vector

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.

这个子项目是许多利用资源的研究子项目之一 由NIH/NCRR资助的中心拨款提供。子项目的主要支持 而子项目的主要调查员可能是由其他来源提供的， 包括其他NIH来源。 列出的子项目总成本可能 代表子项目使用的中心基础设施的估计数量， 而不是由NCRR赠款提供给子项目或子项目工作人员的直接资金。 研究领域集中在无病毒的合成载体用于治疗传递 应用，特别是使用基于阳离子脂质体的载体，最近经历了一个 文艺复兴。这是由于载体的非免疫原性和免疫原性的潜力。 将大的DNA片段，例如约1 Mbps的人类人工DNA片段， 这对于病毒载体是不可行的。然而，转染 效率（TE;外源基因的表达的量度，所述外源基因被转移到细胞中， 细胞）的阳离子脂质-DNA复合物（CLDC），与病毒载体相比仍然较低， 只有我们对CLDC之间相互作用的了解进一步显著增加， 细胞将导致TE的体外以及体内优化。