siRNA 药物递送至阿尔茨海默病的血脑屏障。
siRNA drug delivery across the blood-brain barrier in Alzheimer's disease.
机构信息
Department of Biomedical and Pharmaceutical Sciences, Harry and Diane Rinker Health Science Campus, Chapman University School of Pharmacy, Irvine, CA 92618, USA; Faculty of Pharmacy, University of Central Punjab, Lahore 54000, Pakistan.
Shifa College of Pharmaceutical Sciences, Shifa Tameer-e-Millat University, Islamabad 44000, Pakistan.
出版信息
Adv Drug Deliv Rev. 2023 Aug;199:114968. doi: 10.1016/j.addr.2023.114968. Epub 2023 Jun 21.
Alzheimer's disease (AD) is a progressive neurodegenerative disease with a few FDA-approved drugs that provide modest symptomatic benefits and only two FDA-approved disease-modifying treatments for AD. The advancements in understanding the causative genes and non-coding sequences at the molecular level of the pathophysiology of AD have resulted in several exciting research papers that employed small interfering RNA (siRNA)-based therapy. Although siRNA is being sought by academia and biopharma industries, several challenges still need to be addressed. We comprehensively report the latest advances in AD pathophysiology, druggable targets, ongoing clinical trials, and the siRNA-based approaches across the blood-brain barrier for addressing AD. This review describes the latest delivery systems employed to address this barrier. Critical insights and future perspectives on siRNA therapy for AD are also provided.
阿尔茨海默病（AD）是一种进行性神经退行性疾病，仅有少数获得 FDA 批准的药物可提供适度的症状缓解，且仅有两种获得 FDA 批准的 AD 疾病修饰治疗方法。在理解 AD 病理生理学的分子水平上的致病基因和非编码序列方面取得的进展，促成了几篇令人兴奋的研究论文，这些论文采用了小干扰 RNA（siRNA）为基础的治疗方法。尽管学术界和生物制药行业都在寻求 siRNA，但仍有几个挑战需要解决。我们全面报告了 AD 病理生理学、可成药靶点、正在进行的临床试验以及跨越血脑屏障用于治疗 AD 的 siRNA 方法方面的最新进展。本综述描述了为解决这一障碍而采用的最新递药系统。还对 AD 的 siRNA 治疗的关键见解和未来展望进行了阐述。
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