Gene Therapy Offers Potential Treatment Approach for Mitochondrial Diseases

Scientists say they have developed a genome editing tool for the potential treatment of mitochondrial diseases, which are serious and often fatal conditions affecting 1 in 5000 people.The team, led by the University of Cambridge, applied an experimental gene therapy treatment in mice, and were able to successfully target and eliminate the damaged DNA in mitochondria which causes the devastating conditions.  The study (“ Genome editing in mitochondria corrects a pathogenic mtDNA m