Yuchuan Yang , Xiaoying Xu

Biotechnology Research Center, Cuixi Academy of Biotechnology, Zhuji, 311800, China
Author    Correspondence author
International Journal of Molecular Medical Science, 2024, Vol. 14, No. 4   
Received: 08 Jul., 2024    Accepted: 10 Aug., 2024    Published: 23 Aug., 2024

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.

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.

Alzheimer's disease; Gene therapy; CRISPR/Cas9; Amyloid-β; Personalized medicine