Gene therapy for mitochondrial DNA defects: is it possible?

Article

Gene therapy for mitochondrial DNA defects: is it possible?

Gene therapy - 1 Jul 1995

Chrzanowska-Lightowlers Z M, Lightowlers R N, Turnbull D M

Abstract excerpt

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 gradua...

Topics

DNA, Mitochondrial
Genetic Therapy
Humans
Metabolism, Inborn Errors
Mutation

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