Department of Biomedical Engineering, Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, Wisconsin 53715, USA.
Department of Biomedical Engineering and Center for Advanced Genomic Technologies, Duke University, Durham, North Carolina 27708, USA; email:
Annu Rev Biomed Eng. 2021 Jul 13;23:493-516. doi: 10.1146/annurev-bioeng-122019-121602. Epub 2021 Apr 28.
The recent discovery and subsequent development of the CRISPR-Cas9 (clustered regularly interspaced short palindromic repeat-CRISPR-associated protein 9) platform as a precise genome editing tool have transformed biomedicine. As these CRISPR-based tools have matured, multiple stages of the gene editing process and the bioengineering of human cells and tissues have advanced. Here, we highlight recent intersections in the development of biomaterials and genome editing technologies. These intersections include the delivery of macromolecules, where biomaterial platforms have been harnessed to enable nonviral delivery of genome engineering tools to cells and tissues in vivo. Further, engineering native-like biomaterial platforms for cell culture facilitates complex modeling of human development and disease when combined with genome engineering tools. Deeper integration of biomaterial platforms in these fields could play a significant role in enabling new breakthroughs in the application of gene editing for the treatment of human disease.
最近发现并随后开发的 CRISPR-Cas9（成簇规律间隔短回文重复序列 -CRISPR 相关蛋白 9）平台作为一种精确的基因组编辑工具，已经改变了生物医学。随着这些基于 CRISPR 的工具的成熟，基因编辑过程的多个阶段以及人类细胞和组织的生物工程都取得了进展。在这里，我们重点介绍生物材料和基因组编辑技术的最新交叉点。这些交叉点包括大分子的传递，其中生物材料平台已被利用来实现非病毒将基因组工程工具递送到体内细胞和组织。此外，当与基因组工程工具结合使用时，用于细胞培养的天然样生物材料平台的工程化有助于复杂的人类发育和疾病建模。在这些领域中，更深入地整合生物材料平台可能会在基因编辑治疗人类疾病的应用中发挥重要作用，从而实现新的突破。
