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Lipid nanoparticle mediated small interfering RNA delivery as a potential therapy for Alzheimer's disease
European Journal of Neroscience ( IF 3.4 ) Pub Date : 2024-04-16 , DOI: 10.1111/ejn.16336
Tanvir Ahmed 1
Affiliation  

Alzheimer's disease (AD) is a neurodegenerative condition that exhibits a gradual decline in cognitive function and is prevalent among a significant number of individuals globally. The use of small interfering RNA (siRNA) molecules in RNA interference (RNAi) presents a promising therapeutic strategy for AD. Lipid nanoparticles (LNPs) have been developed as a delivery vehicle for siRNA, which can selectively suppress target genes, by enhancing cellular uptake and safeguarding siRNA from degradation. Numerous research studies have exhibited the effectiveness of LNP‐mediated siRNA delivery in reducing amyloid beta (Aβ) levels and enhancing cognitive function in animal models of AD. The feasibility of employing LNP‐mediated siRNA delivery as a therapeutic approach for AD is emphasized by the encouraging outcomes reported in clinical studies for other medical conditions. The use of LNP‐mediated siRNA delivery has emerged as a promising strategy to slow down or even reverse the progression of AD by targeting the synthesis of tau phosphorylation and other genes linked to the condition. Improvement of the delivery mechanism and determination of the most suitable siRNA targets are crucial for the efficacious management of AD. This review focuses on the delivery of siRNA through LNPs as a promising therapeutic strategy for AD, based on the available literature.

中文翻译:

脂质纳米颗粒介导的小干扰RNA递送作为阿尔茨海默病的潜在疗法

阿尔茨海默病 (AD) 是一种神经退行性疾病,表现为认知功能逐渐下降,在全球大量人群中普遍存在。在 RNA 干扰 (RNAi) 中使用小干扰 RNA (siRNA) 分子为 AD 提供了一种有前景的治疗策略。脂质纳米颗粒 (LNP) 已被开发为 siRNA 的递送载体,它可以通过增强细胞摄取并防止 siRNA 降解来选择性抑制靶基因。大量研究表明,LNP 介导的 siRNA 递送可有效降低 AD 动物模型中的淀粉样蛋白 (Aβ) 水平并增强认知功能。其他医学病症的临床研究中报告的令人鼓舞的结果强调了采用 LNP 介导的 siRNA 递送作为 AD 治疗方法的可行性。 LNP 介导的 siRNA 递送的使用已成为一种有前途的策略,通过靶向 tau 磷酸化和与该疾病相关的其他基因的合成来减缓甚至逆转 AD 的进展。改进递送机制和确定最合适的 siRNA 靶点对于 AD 的有效治疗至关重要。本综述基于现有文献,重点关注通过 LNP 递送 siRNA 作为一种有前景的 AD 治疗策略。
更新日期:2024-04-16
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