Department of Human Genetics and Biochemistry, Sackler School of Medicine, Felsenstein Medical Research Center, Tel Aviv University, Tel Aviv 69978, Israel.
Department of Human Genetics and Biochemistry, Sackler School of Medicine, Felsenstein Medical Research Center, Tel Aviv University, Tel Aviv 69978, Israel.
Adv Drug Deliv Rev. 2022 Nov;190:114535. doi: 10.1016/j.addr.2022.114535. Epub 2022 Sep 19.
Tissue-specific uptake and sufficient biodistribution are central goals in drug development. Crossing the blood-brain barrier (BBB) represents a major challenge in delivering therapeutics to the central nervous system (CNS). Since its discovery in the late 19th century, considerable efforts have been invested in an attempt to decipher the BBB structure complexity and plasticity. In parallel, another prevalent approach is to improve a delivery system by harnessing the biological machinery in an attempt to enhance therapeutic-agent permeability. Here, we review the advantages and limitations of using extracellular vesicles over AAV systems as a delivery system for therapy, focusing on neurodevelopmental disorders.
组织特异性摄取和足够的生物分布是药物开发的核心目标。穿过血脑屏障 (BBB) 是将治疗药物递送到中枢神经系统 (CNS) 的主要挑战。自 19 世纪末发现以来,人们投入了大量精力试图破解 BBB 的结构复杂性和可塑性。与此同时,另一种流行的方法是利用生物机制来改进递药系统,试图提高治疗剂的通透性。在这里,我们回顾了使用细胞外囊泡而非 AAV 系统作为递药系统治疗神经发育障碍的优缺点。