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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Turnbull, D., Lightowlers, R. A roundabout route to gene therapy. Nat Genet 30, 345–346 (2002). https://doi.org/10.1038/ng0402-345
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DOI: https://doi.org/10.1038/ng0402-345
