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BASE EDITORS, COMPOSITIONS, AND METHODS FOR MODIFYING THE MITOCHONDRIAL GENOME

机译:基础编辑器,组合物和修饰线粒体基因组的方法

摘要

The specification provides programmable base editors that are capable of introducing a nucleotide change and/or which could alter or modify the nucleotide sequence at a target site in mitochondrial DNA (mtDNA) with high specificity and efficiency. Moreover, the disclosure provides fusion proteins and compositions comprising a programmable DNA binding protein (e.g., a mitoTALE, a mitoZFP, or a CRISPR/Casp) and double-stranded DNA deaminase that is capable of being delivered to the mitochondria and carrying out precise installation of nucleotide changes in the mtDNA. The fusion proteins and compositions are not limited for use with mtDNA, but also may be used for base editing of any double- stranded target DNA.
机译:该说明书提供了能够引入核苷酸变化和/或可以以具有高特异性和效率改变或修改靶位点的核苷酸序列的可编程基础编辑器。此外,本公开内容提供了包含可编程DNA结合蛋白的融合蛋白和组合物(例如,例如,Mitootale,MitozFP或CRISPR / CASP)和能够递送的双链DNA脱氨酶对线粒体进行精确安装MTDNA的核苷酸变化。融合蛋白和组合物不受限制不限于MTDNA,而且还可用于任何双链靶DNA的基础编辑。

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