Base editing: a brief review and a practical example
Dongwook C.Choe
Kiran Musunuru
Division of Cardiology and Cardiovascular Institute, Department of Medicine, Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA
摘要：Genome editing has undergone rapid development in recent years,yielding new approaches to make precise changes in genes.In this review,we discuss the development of various adenine and cytosine base-editing technologies,which share the ability to make specific base changes at specific sites in the genome.We also describe multiple applications of base editing in vitro and in vivo.Finally,as a practical example,we demonstrate the use of a cytosine base editor and an adenine base editor in human cells to introduce and then correct a prevalent mutation responsible for hereditary tyrosinemia type 1.
机标关键词：
分类号：Q789(基因工程（遗传工程）)
论文发表日期：2021-01-01
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：8( 107-114 )
英文信息