Jargalsaikhan Bat-Erdene, Muto Masanaga, Ema Masatsugu
Department of Stem Cells and Human Disease Models, Research Center for Animal Life Science, Shiga University of Medical Science, Seta, Tsukinowa-cho, Otsu 520-2192, Japan.
Medical Genome Center, National Cerebral and Cardiovascular Center, 6-1 Kishibe-Shinmachi, Suita 564-8565, Japan.
Viruses. 2025 Jul 24;17(8):1036. doi: 10.3390/v17081036.
Over 35 years of history, the field of gene therapy has undergone much progress. The initial concept-the replacement of dysfunctional genes with correct ones-has advanced to the next stage and reached the level of precise genome editing. Dozens of gene therapy products based on viral and non-viral delivery platforms have been approved, marking the dawn of the gene therapy era. These viral vector strategies rely on adenoviruses, adeno-associated viruses, lentivirus-derived tools, and so on. From the middle of the gene therapy transition, despite the challenges and serious negative consequences, the lentiviral vector has emerged as a cornerstone and demonstrated benefits in fields ranging from basic science to gene therapy. Therefore, we outline the importance of lentiviral vectors in the gene therapy era by focusing on their roles in the clinical usage, derivation, and development of next-generation platforms, as well as their pseudotyping.
在超过35年的历史中,基因治疗领域取得了很大进展。最初的概念——用正确的基因替换功能失调的基因——已经发展到了下一个阶段,达到了精确基因组编辑的水平。数十种基于病毒和非病毒递送平台的基因治疗产品已获批准,标志着基因治疗时代的曙光。这些病毒载体策略依赖于腺病毒、腺相关病毒、慢病毒衍生工具等。从基因治疗转型中期开始,尽管面临挑战和严重的负面后果,但慢病毒载体已成为基石,并在从基础科学到基因治疗的各个领域都展现出优势。因此,我们通过关注慢病毒载体在临床应用、下一代平台的衍生和开发以及假型化方面的作用,概述其在基因治疗时代的重要性。