Journal Article

Suppression of disease phenotypes of adult mito-mice carrying pathogenic mtDNA by bone marrow transplantation

Shin-Ichi Inoue, Kaori Ishikawa, Kazuto Nakada, Akitsugu Sato, Hiroyuki Miyoshi and Jun-Ichi Hayashi

in Human Molecular Genetics

Volume 15, issue 11, pages 1801-1807
Published in print June 2006 | ISSN: 0964-6906
Published online April 2006 | e-ISSN: 1460-2083 | DOI:

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For directly addressing the issue of gene therapy of adult patients with mitochondrial diseases, we carried out bone marrow transplantation to adult mito-mice with mutated mtDNA and expressing respiration defects for improvement of disease phenotypes. We supposed that bone marrow cells transdifferentiated into various tissues, so that their transplantation would suppress disease phenotypes. The results showed improvement of survival and delayed expression of renal failure. As most mito-mice without a transplant died due to renal failure, we examined whether transplanted bone marrow cells transdifferentiated into renal tissues carrying improved renal function. Histochemical analyses showed that the suppression of disease phenotypes was not due to transdifferentiation, but due to suppression of apoptosis of renal cells. Thus, bone marrow cells possess a novel function of supporting tissues by suppressing apoptosis induced by respiration defects.

Journal Article.  4661 words.  Illustrated.

Subjects: Genetics and Genomics

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