A new breakthrough in gene therapy! Successful treatment of mitochondrial gene mutation disease

Mitochondrial genome mutations (mtDNA) are important causes of mitochondrial disease. Until now, these mitochondrial diseases have remained incurable and difficult to treat effectively due to exosomes, manifestations and prognostic heterogeneity.
to bring new treatments for the disease, scientists from the University of Cambridge recently used a recently developed mouse model (m.5024C>T TRNAAla mice) that represent common molecular characteristics of heterogeneity mtDNA disease in heart tissue. The discovery of gene therapy, using gene editing techniques to successfully eliminate mtDNA mutations in mouse heart tissue, was published recently in Nature Medicine under the title "Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo".
researchers used a programmable nuclease treatment that systematically injected adeno-related viruses to deliver zinc finger nuclease (mtZFN) to heart tissue to the heart tissue, and the researchers succeeded in specifically removing mutant mtDNA from the hearts of mice, along with molecular and biochemical ideopolysis reversals in mice.
findings suggest that gene repair using programmable nuclease-targeted mtDNA has the potential to become a gene therapy for various genetically-caused heterogeneic mitochondrial diseases. (Bio Valley)