Article

Targeted deletions in human mitochondrial DNA engineered by Type V CRISPR-Cas12a system

2024-10-21

Abstract excerpt

Mutations in mitochondrial DNA (mtDNA) contribute to various neuromuscular diseases, with severity depending on heteroplasmy level when mutant and wild-type mtDNA coexist within the same cell. Developing methods to model mtDNA dysfunction is crucial for experimental therapies. Here, we adapted the Type V CRISPR-AsCas12a system, which recognizes AT-rich PAM sequences, for targeted editing of human mtDNA. We show th...

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Literature Corpus work: 1b452b3b-b77f-595f-a9e6-bd3b3ecb5d71
DOI: 10.1101/2024.10.20.619292

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