Article
Manipulation of Murine Mitochondrial DNA Heteroplasmy with mtZFNs.
Methods in molecular biology (Clifton, N.J.) - 1 Jan 2023
Nash Pavel A, Minczuk Michal
Abstract excerpt
Mouse models of mitochondrial DNA mutations hold promise in the development and optimization of mitochondrial gene therapy technology and for gathering pre-clinical data prior to human trials. Their suitability for this purpose stems from the high similarity of human and murine mitochondrial genomes and the increasing availability of rationally designed AAV vectors capable of selectively transducing murine...
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