基因组工程在医学合成生物学中的应用

Application of genome engineering in medical synthetic biology

摘要:

合成生物学旨在建立一套完整的工程理论和方法，通过设计和组装基本生物学元件，更为有效地实现复杂生物系统的设计，并使其完成可编程的生物学功能。近年来随着可编程基因组元件的出现，特别是CRISPR和CRISPRi技术平台的建立和完善，使得合成生物学进入了一个全新发展的时期。本文重点综述CRISPR等基因组编辑和调控技术，其在构建可编程生物学元件和复杂基因线路的应用以及合成生物学在医学中 (称为医学合成生物学) 的发展前景。

Abstract:

Synthetic biology aims to establish a complete set of engineering principles, theories, and methods, via the rational design and assembly of basic biological parts, for the goal of effective implementation of complex biological systems with programmable functions. In recent years, with emerging novel classes of programmable genetic parts, in particular, the establishment and optimization of CRISPR and CRISPRi technology platforms, synthetic biology is entering a new era. This review summarizes recent advances on CRISPR genome editing and gene regulation technologies, their applications in constructing programmable biological parts, and their roles in building sophisticated gene circuits. We also provide a future vision on how synthetic biology can transform medicine (named medical synthetic biology, MSB) and therapeutics.