Xie Mingqi, Viviani Mirta, Fussenegger Martin
Key Laboratory of Growth Regulation and Translational Research of Zhejiang Province, School of Life Sciences, Westlake University, Hangzhou 310024, Zheijang, China.
Westlake Laboratory of Life Sciences and Biomedicine, Hangzhou 310024, Zhejiang, China.
Synth Biol (Oxf). 2020 Nov 24;6(1):ysaa024. doi: 10.1093/synbio/ysaa024. eCollection 2021.
In the past decade, gene- and cell-based therapies have been at the forefront of the biomedical revolution. Synthetic biology, the engineering discipline of building sophisticated 'genetic software' to enable precise regulation of gene activities in living cells, has been a decisive success factor of these new therapies. Here, we discuss the core technologies and treatment strategies that have already gained approval for therapeutic applications in humans. We also review promising preclinical work that could either enhance the efficacy of existing treatment strategies or pave the way for new precision medicines to treat currently intractable human conditions.
在过去十年中，基于基因和细胞的疗法一直处于生物医学革命的前沿。合成生物学作为构建复杂“基因软件”以实现对活细胞中基因活动进行精确调控的工程学科，已成为这些新疗法取得决定性成功的因素。在此，我们讨论已获批准用于人类治疗应用的核心技术和治疗策略。我们还回顾了有前景的临床前研究工作，这些工作要么可以提高现有治疗策略的疗效，要么为治疗当前难以治愈的人类疾病的新型精准药物铺平道路。
