Both teams are reportedly planning to launch clinical trials, Science reports. Researchers have successfully corrected disease-causing mitochondrial mutations in live mice with one different gene editing approaches. In a the University of Cambridge study, adeno-associated viruses containing transcription activator-like effector nucleases (TALENs) 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 Minnesota -based study at University of Miami injected the tail veins of mice with mitochondrial-targeted zinc-finger nucleases (ZFNs), which led to 50% 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 one different gene editing approaches. In a the University of Cambridge study, adeno-associated viruses containing transcription activator-like effector nucleases (TALENs) 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 Minnesota -based study at University of Miami injected the tail veins of mice with mitochondrial-targeted zinc-finger nucleases (ZFNs), which led to 50% less mutant mtDNA in cardiac tissue. The test successes suggest an application to humans may be possible.