Litcius/Paper detail

Editing the Mitochondrial Genome

Maria Falkenberg, Michio Hirano

2020New England Journal of Medicine14 citationsDOIOpen Access PDF

Abstract

For most patients with mitochondrial disorders, most of which are caused by mutations in genes in the mitochondrial genome, only symptomatic treatment is available. A recently published report describes a proof-of-principle study involving the enzymatic editing of mitochondrial DNA.

Topics & Concepts

Mitochondrial DNAMitochondrial diseaseMitochondrionGenome editingGenomeGeneGeneticsRNA editingHuman mitochondrial geneticsMedicineComputational biologyBiologyRNAMitochondrial Function and PathologyMetabolism and Genetic DisordersCRISPR and Genetic Engineering