International Journal of Molecular Medical Science, 2024, Vol.14, No.4, 252-263 http://medscipublisher.com/index.php/ijmms 252 Review and Progress Open Access Prospects of Gene Therapy in Alzheimer's Disease Yuchuan Yang, Xiaoying Xu Biotechnology Research Center, Cuixi Academy of Biotechnology, Zhuji, 311800, Zhejiang, China Corresponding author: xiaoying.xu@cuixi.org International Journal of Molecular Medical Science, 2024, Vol.14, No.4 doi: 10.5376/ijmms.2024.14.0027 Received: 08 Jul., 2024 Accepted: 10 Aug., 2024 Published: 23 Aug., 2024 Copyright © 2024 Yang and Xu, This is an open access article published under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Preferred citation for this article: Yang Y.C., and Xu X.Y., 2024, Prospects of gene therapy in Alzheimer's disease, International Journal of Molecular Medical Science, 14(4): 252-263 (doi: 10.5376/ijmms.2024.14.0027) Abstract Alzheimer's disease (AD) is a progressive neurodegenerative disorder primarily driven by the accumulation of amyloid-β plaques and neurofibrillary tangles. Despite advances in understanding its molecular mechanisms, current treatments remain largely symptomatic. Gene therapy, by targeting specific genetic mutations and pathological pathways, offers a promising approach to addressing the root causes of AD. This study explores the potential of gene therapy in AD, with a focus on the latest advancements in gene editing technologies, such as CRISPR/Cas9, the development of innovative gene delivery systems, and the rise of personalized medicine. The study also discusses the challenges of clinical translation for gene therapy, including technical, ethical, and regulatory hurdles. Finally, the research highlights future directions in gene therapy, particularly the exploration of new therapeutic targets beyond amyloid and tau, and the integration of gene therapy with other treatment modalities to achieve synergistic effects. Despite significant challenges, the prospects of gene therapy in AD offer great hope for fundamentally altering the treatment landscape of this disease. Keywords Alzheimer's disease; Gene therapy; CRISPR/Cas9; Amyloid-β; Personalized medicine 1 Introduction Alzheimer's disease (AD) is a progressive neurodegenerative disorder that stands as the most common cause of dementia, affecting millions of individuals worldwide. Characterized by cognitive decline, memory loss, and behavioral changes, AD imposes a significant burden not only on patients but also on caregivers and healthcare systems globally. The pathophysiology of AD is complex, involving the accumulation of amyloid-beta (Aβ) plaques, the formation of neurofibrillary tangles composed of hyperphosphorylated tau protein, and widespread neuronal loss. Despite extensive research and the development of various therapeutic strategies, there remains no cure for AD, and current treatments are primarily symptomatic, providing only temporary relief without altering the disease's course (Weber-Adrian, 2019; Owens et al., 2021). The progressive nature of AD and the lack of effective disease-modifying treatments underscore the urgent need for innovative therapeutic approaches (Ghaffari et al., 2020). Given the limitations of existing therapies, there is a growing interest in novel approaches that target the underlying molecular and genetic mechanisms of AD. Gene therapy, which involves the modification or manipulation of gene expression within a patient's cells, offers a promising avenue for the treatment of AD. Unlike conventional therapies that focus on alleviating symptoms, gene therapy aims to correct or modulate the genetic defects and pathological processes that drive the disease. This approach has the potential to not only slow the progression of AD but also to address the root causes of the disorder (Loera-Valencia et al., 2018; Sudhakar and Richardson, 2018). Advances in gene delivery systems, including viral and non-viral vectors, along with breakthroughs in gene-editing technologies like CRISPR/Cas9, have paved the way for innovative gene therapy strategies that could revolutionize the treatment of AD (Rudenko and Sholomon, 2023; Unnisa et al., 2023). This study will provide a comprehensive analysis of the current status and future prospects of gene therapy in the treatment of Alzheimer's disease (AD). It will explore the genetic basis of AD, the mechanisms and targets of gene therapy, and the current therapeutic strategies. The study will also discuss ongoing research and clinical trials, addressing the challenges and limitations of gene therapy in this context. By synthesizing the latest research and insights, this study will offer a detailed explanation of the critical role that gene therapy plays in tackling Alzheimer's disease.
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