Lollo C P, Banaszczyk M G, Chiou H C
Immune Response Corporation, 5935 Darwin Court, Carisbad, CA 92008, USA.
Curr Opin Mol Ther. 2000 Apr;2(2):136-42.
This review focuses on recent progress and novel strategies to improve the efficiency of in vivo non-viral gene delivery. Examples of the most promising attempts to overcome specific barriers are presented in fuller detail. Current research into several of the most difficult steps in the gene delivery pathway is discussed including particle stabilization, targeting, cytoplasmic entry and access to the nucleus. The impact of recent reports on our current understanding of the true limitations to in vivo delivery is also discussed. The importance of preclinical animal models for the development of clinical applications of gene therapy is noted.
本综述聚焦于提高体内非病毒基因递送效率的最新进展和新策略。更详细地介绍了克服特定障碍的最有前景的尝试实例。讨论了目前对基因递送途径中几个最困难步骤的研究,包括颗粒稳定、靶向、细胞质进入和细胞核进入。还讨论了近期报告对我们目前对体内递送真正限制的理解的影响。指出了临床前动物模型对基因治疗临床应用开发的重要性。