拓展基因编辑工具和靶点。
Expanding the Toolbox and Targets for Gene Editing.
机构信息
Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA.
Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA.
出版信息
Trends Mol Med. 2021 Mar;27(3):203-206. doi: 10.1016/j.molmed.2020.12.005. Epub 2021 Jan 21.
Genome editing holds great promise for treating a range of human genetic diseases. While emerging clustered regularly interspaced short-palindromic repeats (CRISPR) technologies allow editing of the nuclear genome, it is still not possible to precisely manipulate mitochondrial DNA (mtDNA). Here, we summarize past developments and recent advances in nuclear and mitochondrial genome editing.
基因组编辑在治疗一系列人类遗传疾病方面具有巨大的潜力。虽然新兴的成簇规律间隔短回文重复序列（CRISPR）技术允许对核基因组进行编辑，但仍然不可能精确地操作线粒体 DNA（mtDNA）。在这里，我们总结了核基因组和线粒体基因组编辑的过去发展和最新进展。
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