siRNA drug delivery across the blood-brain barrier in Alzheimer's Disease

Adv Drug Deliv Rev. 2023 Jun 21:114968. doi: 10.1016/j.addr.2023.114968. Online ahead of print.ABSTRACTAlzheimer'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.PMID:37353152 | DOI:10.1016/j.addr.2023.114968
Source: Advanced Drug Delivery Reviews - Category: Drugs & Pharmacology Authors: Source Type: research