DiCarlo James E, Deeconda Anurag, Tsang Stephen H
Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA.
Department of Pathology and Cell Biology, Institute of Human Nutrition, College of Physicians, Columbia University, New York, NY, USA.
Adv Exp Med Biol. 2017;1016:3-27. doi: 10.1007/978-3-319-63904-8_1.
Over the past few decades the ability to edit human cells has revolutionized modern biology and medicine. With advances in genome editing methodologies, gene delivery and cell-based therapeutics targeted at treatment of genetic disease have become a reality that will become more and more essential in clinical practice. Modifying specific mutations in eukaryotic cells using CRISPR-Cas systems derived from prokaryotic immune systems has allowed for precision in correcting various disease mutations. Furthermore, delivery of genetic payloads by employing viral tropism has become a crucial and effective mechanism for delivering genes and gene editing systems into cells. Lastly, cells modified ex vivo have tremendous potential and have shown effective in studying and treating a myriad of diseases. This chapter seeks to highlight and review important progress in the realm of the editing of human cells using CRISPR-Cas systems, the use of viruses as vectors for gene therapy, and the application of engineered cells to study and treat disease.
在过去几十年里，编辑人类细胞的能力彻底改变了现代生物学和医学。随着基因组编辑方法的进步，针对遗传疾病治疗的基因递送和基于细胞的疗法已成为现实，并且在临床实践中将变得越来越重要。利用源自原核免疫系统的CRISPR-Cas系统修饰真核细胞中的特定突变，已能够精确校正各种疾病突变。此外，利用病毒嗜性递送遗传物质已成为将基因和基因编辑系统递送至细胞的关键且有效机制。最后，体外修饰的细胞具有巨大潜力，并且已在研究和治疗多种疾病中显示出有效性。本章旨在突出并回顾在使用CRISPR-Cas系统编辑人类细胞领域、将病毒用作基因治疗载体以及工程细胞在研究和治疗疾病中的应用方面取得的重要进展。
