Smyth Templeton Nancy
Center for Cell and Gene Therapy, Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, Texas, USA.
DNA Cell Biol. 2002 Dec;21(12):857-67. doi: 10.1089/104454902762053828.
Optimization of cationic liposomal complexes for in vivo applications and therapeutics is complex involving many distinct components. These components include nucleic acid purification, plasmid design, formulation of the delivery vehicle, administration route and schedule, dosing, detection of gene expression, and others. This review will focus on optimization of these components for use in a variety of in vivo applications. Use of improved liposome formulations for delivery in vivo is valuable for gene therapy and would avoid several problems associated with viral delivery. Delivery of nucleic acids using liposomes is promising as a safe and non-immunogenic approach to gene therapy. Furthermore, gene therapeutics composed of artificial reagents can be standardized and regulated as drugs rather than as biologics. Optimizing all components of the delivery system will allow broad use of liposomal complexes to treat or cure human diseases or disorders.
用于体内应用和治疗的阳离子脂质体复合物的优化过程很复杂,涉及许多不同的组成部分。这些组成部分包括核酸纯化、质粒设计、递送载体的配方、给药途径和时间表、剂量、基因表达检测等。本综述将重点关注这些组成部分在各种体内应用中的优化。使用改进的脂质体制剂进行体内递送对于基因治疗很有价值,并且可以避免与病毒递送相关的几个问题。使用脂质体递送核酸作为一种安全且无免疫原性的基因治疗方法很有前景。此外,由人工试剂组成的基因治疗剂可以作为药物而不是生物制品进行标准化和监管。优化递送系统的所有组成部分将使脂质体复合物能够广泛用于治疗或治愈人类疾病或病症。