Alzheimer’s disease (AD), a neuro-degenerative disease that primarily affects the elderly, is a worldwide phenomenon. Loss of memory, cognitive decline, behavioural changes, and many other signs are used to classify it. Various hypotheses that may contribute to Alzheimer’s disease have been found during decades of survey, including tau theory, the amyloid theory, the cholinergic hypothesis, and the oxidative stress hypothesis. According to some theories, the two leading causes of AD are the accumulation of amyloid beta plaque and development of NFTs in the brain. The hippocampus and cerebral cortex are the primary sites where amyloid beta plaques gather in the body. NFT formation in the brain impairs the brain’s neurons’ potential of signalling. According to the age at which it manifests in a person, there are two subtypes of AD: ‘LOAD (Late Onset Alzheimer’s Disease)’ and ‘EOAD (Early Onset Alzheimer’s Disease)’. Long-term research into AD treatment has resulted in the introduction of some medications that provided symptomatic relief to patients but did not alter the disease’s pathophysiology, like cholinesterase inhibitors, inhibitors of tau aggregation, and monoclonal antibodies to Aβ aggregation. Even though the medications did not halt the progression of AD, researchers did not discontinue their work, which lead to the introduction of gene therapy — a recently created cutting-edge method of delivering genes to target sites where they can express the intended functionalities. Viral or non-viral vectors could be used to deliver the gene, each with advantages and limitations of their own. Gene therapy is proven to be a potential disease-modifying treatment for AD. This article discusses about gene therapy, its merits and demerits and the various ways of gene delivery. Additionally, it focuses on AD as the target for treatment through gene therapy, the pathophysiology of AD, and the multiple targets for gene therapy in the treatment of AD.

阿尔茨海默病 (AD) 是一种主要影响老年人的神经退行性疾病，是一种世界性现象。记忆丧失、认知能力下降、行为改变和许多其他迹象都被用来对其进行分类。经过数十年的研究，人们发现了可能导致阿尔茨海默病的各种假说，包括 tau 蛋白假说、淀粉样蛋白假说、胆碱能假说和氧化应激假说。根据一些理论，AD 的两个主要原因是大脑中β淀粉样蛋白斑块的积累和 NFT 的形成。海马体和大脑皮层是体内β淀粉样蛋白斑块聚集的主要部位。大脑中 NFT 的形成会损害大脑神经元的信号传导潜力。根据人出现的年龄，AD 有两种亚型：“LOAD（晚发性阿尔茨海默病）”和“EOAD（早发性阿尔茨海默病）”。对 AD 治疗的长期研究导致引入了一些药物，这些药物可以缓解患者的症状，但不会改变疾病的病理生理学，例如胆碱酯酶抑制剂、tau 蛋白聚集抑制剂和抗 Aβ 聚集的单克隆抗体。尽管这些药物并没有阻止 AD 的进展，但研究人员并没有停止他们的工作，这导致了基因疗法的引入——一种最近创建的尖端方法，将基因传递到可以表达预期功能的目标位点。病毒或非病毒载体可用于传递基因，每种载体都有各自的优点和局限性。基因疗法被证明是一种潜在的 AD 疾病缓解疗法。本文讨论了基因治疗、其优点和缺点以及基因递送的各种方式。 此外，它还重点关注AD作为基因治疗的治疗靶点、AD的病理生理学以及基因治疗在AD治疗中的多个靶点。