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 technologi...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.展开更多
基金This work was supported by U.S.National Institutes of Health grant R35-HL145203 and the Winkelman Family Fund in Cardiovascular Innovation(K.M.).K.M.is a co-founder and advisor of Verve Therapeutics and an advisor of Variant Bio.
文摘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.
