Approximately one in 200 women carry defective mitochondrial DNA (mtDNA), and their children can develop serious or fatal diseases. "Back in 2008 we provided a proof of principle for the gene therapy approach when we were able to shift a balance between diseased and normal mtDNA in cells by targeting a restriction endonuclease - an enzyme that cuts DNA at a predetermined sequence - to mitochondria," Dr. Alexeyev said.
A group of researchers at the University of Miami led by Dr. Carlos Moraes adapted components of the system that pathogenic bacteria use to improve their “living conditions” inside plant cells to develop genetic tools for targeting diseased mtDNA in a broader range of mitochondrial diseases.
The article describes how a team led by developmental biologist Juan Carlos Izpisua Belmonte of the Salk Institute for Biological Studies in San Diego took Dr. Alexeyev's and Dr. Moraes' approach one step further.
Click here to view the full story.
