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Lookup NU author(s): Emeritus Professor Doug Turnbull,
Professor Robert Lightowlers
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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.
Author(s): Turnbull DM, Lightowlers RN
Publication type: Note
Publication status: Published
Journal: Nature Genetics
Print publication date: 01/04/2002
ISSN (print): 1061-4036
ISSN (electronic): 1546-1718
PubMed id: 11925553