Pathogenic mitochondrial DNA(mtDNA)mutations can cause a variety of human diseases.The recent development of genome-editing technologies to manipulate mtDNA,such as mitochondria-targeted DNA nucleases and base editors...Pathogenic mitochondrial DNA(mtDNA)mutations can cause a variety of human diseases.The recent development of genome-editing technologies to manipulate mtDNA,such as mitochondria-targeted DNA nucleases and base editors,offer a promising way for curing mitochondrial diseases caused by mtDNA mutations.The CRISPR-Cas9 system is a widely used tool for genome editing;however,its application in mtDNA editing is still under debate.In this study,we developed a mito-Cas9 system by adding the mitochondria-targeted sequences and 30 untranslated region of nuclear-encoded mitochondrial genes upstream and downstream of the Cas9 gene,respectively.展开更多
基金supported by the National Science and Technology Innovation 2030 Major Program(2021ZD0200900)the National Natural Science Foundation of China(U1702284 and 31970560),Yunnan Province(202003AD150009 and 2019FA027)the Strategic Priority Research Program(B)of the Chinese Academy of Sciences(CAS)(XDB32020200).
文摘Pathogenic mitochondrial DNA(mtDNA)mutations can cause a variety of human diseases.The recent development of genome-editing technologies to manipulate mtDNA,such as mitochondria-targeted DNA nucleases and base editors,offer a promising way for curing mitochondrial diseases caused by mtDNA mutations.The CRISPR-Cas9 system is a widely used tool for genome editing;however,its application in mtDNA editing is still under debate.In this study,we developed a mito-Cas9 system by adding the mitochondria-targeted sequences and 30 untranslated region of nuclear-encoded mitochondrial genes upstream and downstream of the Cas9 gene,respectively.
