Review Article

外显子在治疗中的工程、药物动力学及应用前景

卷 20, 期 1, 2019

页: [87 - 95] 页: 9

弟呕挨: 10.2174/1389450119666180521100409

价格: $65

摘要

背景:真核细胞在生理和病理条件下释放不同大小的囊泡。细胞外小泡可分为凋亡小体、微囊泡和外小体。其中,外胚层被认为是创新治疗干预的工具,特别是当使用效应分子进行工程时。外胚体的传递功能受到许多典型特征的青睐。它们包括体积小(50~200 nm)、膜成分与产生细胞紧密相似、缺乏毒性、血清和其他生物液体的稳定性以及几乎任何器官和组织,包括中枢神经系统。然而,一些尚未解决的问题仍然影响到在治疗中可能使用外显子。其中包括对体外和体内产生的小泡的精确鉴定、它们的大规模生产和纯化、治疗大分子的上传效率以及它们的药代动力学特征。目的:本文从工程技术和体内生物分布/药代动力学两个方面讨论了将外体作为理想治疗分子的载体进行研究的两个关键问题。此外,还将讨论一种旨在克服至少部分障碍的创新办法,以便在治疗中安全有效地使用外植体。结论:多种生物学特征使外胚层成为治疗分子传递的一种有吸引力的工具。一旦少数尚未克服的技术障碍得到克服,它们肯定会成为创新治疗干预措施的一部分。

关键词: 外体,生物分布,药动学,RNA传递,嵌合蛋白,多囊体。

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