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 (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. Researchers in the US and Europe have used two gene-editing approaches to correct mitochondrial mutations in live mice. The researchers also used TALEN inhibitors to correct genetic defects caused by other mutations, including one linked to cystic fibrosis. 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 (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. The researchers also used TALEN inhibitors to correct genetic defects caused by other mutations, including one linked to cystic fibrosis. The test successes suggest an application to humans may be possible.