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A roundabout route to gene therapy

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.

Publication metadata

Author(s): Turnbull DM, Lightowlers RN

Publication type: Note

Publication status: Published

Journal: Nature Genetics

Year: 2002

Volume: 30

Issue: 4

Pages: 345-346

Print publication date: 01/04/2002

ISSN (print): 1061-4036

ISSN (electronic): 1546-1718


DOI: 10.1038/ng0402-345

PubMed id: 11925553