Tehreem Mushtaq, Huma Hameed, Ana Cláudia Paiva-Santos, Urwa Tariq, Anam Hameed
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引用次数: 0
摘要
阿尔茨海默病(AD)是一种神经退行性疾病,其特征是淀粉样蛋白- β (a β)斑块沉积和tau蛋白异常,破坏突触功能并导致进行性认知能力下降。然而,目前的治疗方法仍然阻碍了研究的重大努力,这些治疗方法受到血脑屏障渗透性差和非特异性作用的限制。纳米技术和药物递送的最新发展发现外泌体是靶向Aβ的创新载体。他们已经确定了一种治疗AD潜在病理的新方法。外泌体是自然发生的细胞外囊泡,具有几个独特的优点。它们具有生物相容性,可以穿过血脑屏障,并且可以精确地传递治疗药物。这些药物包括小干扰RNA (siRNA)、多肽或旨在抑制Aβ聚集、增强其清除或调节其产生相关基因的药物。在这些药物中,神经来源的外泌体提供了很大的希望,因为它们自然地吸引神经元组织,因此增加了治疗的特异性。在临床前研究中,这种疗法已被证明是令人鼓舞的,在AD模型中显示出减少a β积累,减少神经炎症和认知改善。然而,转化为临床应用面临着一些挑战,例如开发可扩展的外泌体生产方法,药物装载效率,稳定性和给药安全性。本文综述了通过外泌体靶向Aβ病理这一日益增长的领域的潜在益处、最近的突破和开放的挑战。利用外泌体的疗法可以创造出治疗阿尔茨海默病的突破性疗法,希望数百万人能从这种毁灭性的疾病中解脱出来。
Exosome-Mediated Delivery of Amyloid Beta Modulators: A Potential Therapeutic Strategy for Alzheimer's Disease.
Alzheimer's disease (AD) is a neurodegenerative disorder characterized by the deposition of amyloid-beta (Aβ) plaques and tau protein abnormalities, disrupting synaptic function and causing progressive cognitive decline. However, significant efforts in research are still hampered by current treatments, which are limited by poor penetration of the BBB and non-specific effects. Recent developments in nanotechnology and drug delivery have found exosomes as innovative carriers targeting Aβ. They have identified a novel approach to treating the underlying pathology of AD. Exosomes are naturally occurring extracellular vesicles with several unique advantages. They are biocompatible, can cross the BBB, and can be engineered to deliver therapeutic agents with precision. These agents range from small interfering RNA (siRNA), peptides, or drugs designed to either inhibit Aβ aggregation, enhance its clearance, or regulate the genes involved in its production. Among these agents, neural-derived exosomes offer great promise as they naturally attract neuronal tissue and, therefore, increase the specificity of the treatment. In preclinical studies, such therapies have proven encouraging by demonstrating reduced Aβ accumulation, a decrease in neuroinflammation, and cognitive improvement in models of AD. However, translation into clinical application faces some challenges, such as development of scalable methods of exosome production, drug loading efficiency, stability, and safety upon administration. The present review takes an outlook toward the growing area of targeting Aβ pathology via exosomes with potential benefits, recent breakthroughs, and open challenges. Harnessed therapy from exosomes can create groundbreaking-therapies in treating AD that hope for millions to come out from this devastator disease.
期刊介绍:
Molecular Neurobiology is an exciting journal for neuroscientists needing to stay in close touch with progress at the forefront of molecular brain research today. It is an especially important periodical for graduate students and "postdocs," specifically designed to synthesize and critically assess research trends for all neuroscientists hoping to stay active at the cutting edge of this dramatically developing area. This journal has proven to be crucial in departmental libraries, serving as essential reading for every committed neuroscientist who is striving to keep abreast of all rapid developments in a forefront field. Most recent significant advances in experimental and clinical neuroscience have been occurring at the molecular level. Until now, there has been no journal devoted to looking closely at this fragmented literature in a critical, coherent fashion. Each submission is thoroughly analyzed by scientists and clinicians internationally renowned for their special competence in the areas treated.