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Gene Therapy for Mitochondrial DNA Defects - is it possible?

Lookup NU author(s): Professor Zofia Chrzanowska-LightowlersORCiD, Professor Robert Lightowlers, Emeritus Professor Doug Turnbull


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Defects of the mitochondrial genome are increasingly being recognised as important causes of disease. Patients may present at any age and the symptoms vary from fatal lactic acidosis in infancy to muscle disease in adults. For most patients there is no satisfactory treatment and there is a gradual deterioration leading to severe disability and death. In the absence of any biochemical treatment, gene therapy must be considered for these patients. This review addresses the unique problems associated with the treatment of defects of the mitochondrial genome by gene therapy, and discusses the approaches which we believe may be of value.

Publication metadata

Author(s): Chrzanowska-Lightowlers ZMA, Lightowlers RN, Turnbull DM

Publication type: Review

Publication status: Published

Journal: Gene Therapy

Year: 1995

Volume: 2

Issue: 5

Pages: 311-316

Print publication date: 01/07/1995

ISSN (print): 0969-7128

ISSN (electronic): 1476-5462