Department of Genetics, Harvard Medical School , Boston, Massachusetts, United States.
Wyss Institute for Biologically Inspired Engineering , Boston, Massachusetts, United States.
ACS Chem Biol. 2018 Feb 16;13(2):313-325. doi: 10.1021/acschembio.7b00842. Epub 2017 Dec 28.
Multiplex genome editing is the simultaneous introduction of multiple distinct modifications to a given genome. Though in its infancy, maturation of this field will facilitate powerful new biomedical research approaches and will enable a host of far-reaching biological engineering applications, including new therapeutic modalities and industrial applications, as well as "genome writing" and de-extinction efforts. In this Perspective, we focus on multiplex editing of large eukaryotic genomes. We describe the current state of multiplexed genome editing, the current limits of our ability to multiplex edits, and provide perspective on the many applications that fully realized multiplex editing technologies would enable in higher eukaryotic genomes. We offer a broad look at future directions, covering emergent CRISPR-based technologies, advances in intracellular delivery, and new DNA assembly approaches that may enable future genome editing on a massively multiplexed scale.
多重基因组编辑是指同时向给定基因组中引入多个不同的修饰。尽管这项技术还处于起步阶段，但该领域的成熟将促进强大的新的生物医学研究方法，并能够实现一系列深远的生物工程应用，包括新的治疗模式和工业应用，以及“基因组编写”和灭绝物种复育工作。在本观点中，我们重点关注大型真核生物基因组的多重编辑。我们描述了多重基因组编辑的现状、目前我们进行多重编辑的能力的限制，并提供了在高等真核生物基因组中充分实现多重编辑技术所能实现的多种应用的视角。我们广泛探讨了未来的发展方向，涵盖了新兴的基于 CRISPR 的技术、细胞内传递的进展以及新的 DNA 组装方法，这些方法可能使未来能够大规模地进行多重基因组编辑。
