Department of Neurology, University of Newcastle upon Tyne, Newcastle upon Tyne, UK. d.m.turnbull@ncl.ac.uk
The uniqueness of the mitochondrial genome presents a number of obstacles to the successful use of gene therapy for the treatment of mitochondrial DNA disease. A new study shows that the effects of a pathogenic mutation in a human mitochondrial gene can be rectified by expressing an engineered wildtype copy of the gene in the nucleus.
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