Gene therapy of the other genome : The challenges of treating mitochondrial DNA defects

Human mitochondrial DNA is a 16.5 kb circular DNA molecule located inside the mitochondrial matrix. Although accounting for only about 1% of total cellular DNA, defects in mitochondrial DNA have been found to have major effects on human health. A single mtDNA mutation may cause a bewildering variety...

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Bibliographic Details
Published in:Pharmaceutical research 2007-02, Vol.24 (2), p.228-238
Main Authors: D'SOUZA, Gerard G. M, BODDAPATI, Sarathi V, WEISSIG, Volkmar
Format: Article
Language:English
Subjects:
Animals
Biological and medical sciences
Circular DNA
DNA - genetics
DNA, Mitochondrial - genetics
Gene therapy
General pharmacology
Genes, Reporter - genetics
Genetic Therapy
Genome
Genomes
Genomics
Humans
Medical sciences
Mitochondrial DNA
Mutation
Mutation - genetics
Neuromuscular system
Pharmaceutical technology. Pharmaceutical industry
Pharmacology
Pharmacology. Drug treatments
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Summary:Human mitochondrial DNA is a 16.5 kb circular DNA molecule located inside the mitochondrial matrix. Although accounting for only about 1% of total cellular DNA, defects in mitochondrial DNA have been found to have major effects on human health. A single mtDNA mutation may cause a bewildering variety of clinical symptoms mainly involving the neuromuscular system at any age of onset. Despite significant advances in the understanding of mitochondrial DNA defects at a molecular level, the clinical diagnosis of mtDNA diseases remains a significant challenge and effective therapies for such diseases are as yet unavailable. In contrast to gene therapy for chromosomal DNA defects, mitochondrial gene therapy is a field that is still in its infancy and attempts towards gene therapy of the mitochondrial genome are rare. In this review we outline what we believe are the unique challenges associated with the correction of mtDNA mutations and summarize current approaches to gene therapy for the "other genome".
ISSN:0724-8741
1573-904X
DOI:10.1007/s11095-006-9150-y