Exosomes in Neurodegenerative Disorders: Isolation Strategies, Therapeutic Potential, and Clinical Trials.

IF 3
Sourin Bhukta, Narahari N Palei, Vishakha Jaiswal, Anuradha Mishra, Prathap M
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Abstract

Exosomes represent a promising class of naturally produced nanoparticles that exist at the nanoscale and carry a negative surface charge under physiological conditions. These tiny membranebound vesicles are released by cells throughout the body and function as biological messengers, transporting various molecular cargos between cells and facilitating critical cell-to-cell communication pathways. Existing therapies for neurodegenerative disorders face two critical barriers: they cannot precisely target the affected brain areas, and the blood-brain barrier blocks most potential treatments from entering the brain. Exosomes offer a promising solution to these challenges. Unlike most synthetic drug delivery systems that struggle to penetrate the brain's protective barrier, these naturally derived nanocarriers exhibit an inherent capacity to traverse the blood-brain barrier. This unique property, combined with their capacity for efficient intracellular delivery of therapeutic payloads, positions exosomes as an exciting platform for transporting pharmaceutical agents to the affected neural tissues. Through strategic engineering and modification, these vesicles can be transformed into highly precise delivery vehicles capable of targeting specific organs, tissues, or even individual cell types. This review explores the therapeutic potential and drug delivery applications of exosomes in the management of major neurodegenerative disorders. This review provides an in-depth examination of exosome biogenesis, current isolation methodologies, and surface engineering strategies, while critically evaluating the strengths and limitations of each approach. In addition, this review summarizes the current preclinical models and provides an overview of ongoing clinical trials investigating exosome-based therapies for neurological disorders.

神经退行性疾病中的外泌体:分离策略、治疗潜力和临床试验。
外泌体代表了一类很有前途的自然产生的纳米颗粒,它们存在于纳米尺度上,在生理条件下携带负电荷。这些微小的膜结合囊泡由全身细胞释放,作为生物信使,在细胞之间运输各种分子货物,促进关键的细胞间通讯途径。现有的神经退行性疾病治疗面临两个关键障碍:它们不能精确地针对受影响的大脑区域,血脑屏障阻止了大多数潜在的治疗进入大脑。外泌体为这些挑战提供了一个有希望的解决方案。与大多数难以穿透大脑保护屏障的合成药物递送系统不同,这些天然衍生的纳米载体表现出固有的穿越血脑屏障的能力。这种独特的特性,加上它们在细胞内有效递送治疗有效载荷的能力,使外泌体成为将药物输送到受影响神经组织的令人兴奋的平台。通过战略性工程和改造,这些囊泡可以转化为高度精确的运载工具,能够靶向特定的器官、组织,甚至单个细胞类型。本文综述了外泌体在治疗主要神经退行性疾病中的治疗潜力和药物传递应用。这篇综述提供了外泌体生物发生、当前分离方法和表面工程策略的深入研究,同时批判性地评估了每种方法的优势和局限性。此外,本综述总结了目前的临床前模型,并概述了正在进行的研究外泌体治疗神经系统疾病的临床试验。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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