Gene therapy of mitochondrial diseases using human cytoplasts
Open Access
- 1 January 1997
- journal article
- review article
- Published by Springer Nature in Gene Therapy
- Vol. 4 (1) , 6-10
- https://doi.org/10.1038/sj.gt.3300347
Abstract
Mitochondrial (mt) DNA is highly susceptible to mutation. A novel approach using cytoplasts is essential for gene therapy of the diseases caused by these mutations, because of their non-Mendelian genetics. In this method, mtDNA is transferred into the mutant cells by forming cybrids and can complement the defect correctly and safely.Keywords
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