Using genome editing to target faulty DNA in mitochondria — the cell's powerhouses — could prevent the inheritance of mitochondrial defects that cause disease.
Juan Carlos Izpisua Belmonte at the Salk Institute for Biological Studies in La Jolla, California, and his colleagues injected RNA into mouse embryos and eggs containing a mix of mitochondria from two different mouse strains. The RNA was programmed to produce an engineered enzyme that targets and cuts DNA from only one mouse strain. Around 60% of the targeted mitochondrial DNA was destroyed.
The researchers also reduced the levels of faulty DNA in mouse eggs that contained mitochondria from humans with a mitochondrial disorder. Eventually, similar techniques might be used to prevent the transmission of such diseases while avoiding a controversial method that makes eggs containing mitochondrial DNA from two women.
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A fix for faulty mitochondria. Nature 521, 8 (2015). https://doi.org/10.1038/521008b
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DOI: https://doi.org/10.1038/521008b
