Researchers have successfully corrected disease - causing mitochondrial mutations in live mice with two different gene editing approaches . Disease-causing mutations in mitochondrial DNA (mtDNA) occur in approximately 1 in 5,000 adults and produce symptoms ranging from muscle weakness to heart disease. In a University of Miami study , adeno - associated viruses containing transcription activator - like effector nucleases ( [UNK] ) were injected into the muscles of mice that had an mtDNA mutation , leading to a 50 % reduction in the levels of the affected material six months later . Meanwhile , a UK - based study at the University of Cambridge injected the tail veins of mice with DNA - targeted zinc - finger nucleases ( ZFNs ) , which led to 40 % less mutant mtDNA in cardiac tissue . The test successes suggest an application to humans may be possible .
Researchers have successfully corrected disease - causing mitochondrial mutations in live mice with two different gene editing approaches . In a University of Miami study , adeno - associated viruses containing transcription activator - like effector nucleases ( [UNK] ) were injected into the muscles of mice that had an mtDNA mutation , leading to a 50 % reduction in the levels of the affected material six months later . Meanwhile , a UK - based study at the University of Cambridge injected the tail veins of mice with DNA - targeted zinc - finger nucleases ( ZFNs ) , which led to 40 % less mutant mtDNA in cardiac tissue . The test successes suggest an application to humans may be possible .