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Article

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

Pharmaceutical research - 1 Feb 2007

D'Souza Gerard G M, Boddapati Sarathi V, Weissig Volkmar

Abstract excerpt

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...

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PMID: 17180727
DOI: 10.1007/s11095-006-9150-y

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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