Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo.

Volume: 24, Issue: 11, Pages: 1691 - 1695
Published: Sep 24, 2018
Abstract
Mutations of the mitochondrial genome (mtDNA) underlie a substantial portion of mitochondrial disease burden. These disorders are currently incurable and effectively untreatable, with heterogeneous penetrance, presentation and prognosis. To address the lack of effective treatment for these disorders, we exploited a recently developed mouse model that recapitulates common molecular features of heteroplasmic mtDNA disease in cardiac tissue: the...
Paper Details
Title
Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo.
Published Date
Sep 24, 2018
Volume
24
Issue
11
Pages
1691 - 1695
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