Gene therapy by mitochondrial transfer

Mitochondrial DNA (mtDNA) is highly susceptible to mutation. Novel approach es such as those involving cytoplast fusion and mitochondrial microinjectio n are essential for gene therapy of diseases caused by these mutations, due to the non-Mendelian genetics of these diseases. In this fusion method, mt DNA in the cytoplast is transferred into mutant cells via the formation of cybrids; once inside the cell the mtDNA complement the defect correctly and safely. The genes in cloned animals are composed of nuclear DNA (nDNA) of a mature tissue and mtDNA from an oocyte. Recent advances in transmitochond rial mice depends on the microinjection of mitochondria into the oocyte. He re we present data on in vitro gene therapy using human mtDNA, cybrid forma tion and microinjection. (C) 2001 Elsevier Science B.V; All rights reserved .