Gene therapy by mitochondrial transfer

Abstract

Mitochondrial DNA (mtDNA) is highly susceptible to mutation. Novel approaches such as those involving cytoplast fusion and mitochondrial microinjection are essential for gene therapy of diseases caused by these mutations, due to the non-Mendelian genetics of these diseases. In this fusion method, mtDNA 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 transmitochondrial mice depends on the microinjection of mitochondria into the oocyte. Here we present data on in vitro gene therapy using human mtDNA, cybrid formation and microinjection.