Yu Jingyu, Li Tianwen, Zhu Jianhong
Department of Neurosurgery, Huashan Hospital, Shanghai Medical College, Fudan University, National Center for Neurological Disorders, National Key Laboratory for Medical Neurobiology, Institutes of Brain Science, Shanghai Key Laboratory of Brain Function and Regeneration, Institute of Neurosurgery, MOE Frontiers Center for Brain Science, Shanghai, China.
Aging Dis. 2023 Apr 1;14(2):398-417. doi: 10.14336/AD.2022.00725.
Rapid advancements have taken place in gene therapy technology. However, effective methods for treating aging- or age-related chronic diseases, which are often closely related to genes or even multiple genes, are still lacking. The path to developing cures is winding, while gene therapy that targets genes related to aging represents an exciting research direction with tremendous potential. Among aging-related genes, some candidates have been studied at different levels, from cell to organismal levels (e.g., mammalian models) with different methods, from overexpression to gene editing. The and have even entered the stage of clinical trials. Even those displaying only a preliminary association with diseases have potential applications. This article discusses the foundations and recent breakthroughs in the field of gene therapy, providing a summary of current mainstream strategies and gene therapy products with clinical and preclinical applications. Finally, we review representative target genes and their potential for treating aging or age-related diseases.
基因治疗技术取得了迅速进展。然而，对于治疗衰老或与年龄相关的慢性疾病，仍然缺乏有效的方法，这些疾病往往与单个基因甚至多个基因密切相关。开发治愈方法的道路曲折，而针对与衰老相关基因的基因治疗是一个令人兴奋且具有巨大潜力的研究方向。在与衰老相关的基因中，一些候选基因已经在从细胞到生物体水平（如哺乳动物模型）的不同层面上，采用从过表达到基因编辑的不同方法进行了研究。甚至已经进入了临床试验阶段。即使那些仅显示与疾病有初步关联的基因也具有潜在应用价值。本文讨论了基因治疗领域的基础和近期突破，总结了当前具有临床和临床前应用的主流策略和基因治疗产品。最后，我们回顾了代表性的靶基因及其治疗衰老或与年龄相关疾病的潜力。
