A novel genetic mitochondrial disorder linked to the LIG3 gene Normally, mitochondrial DNA is repaired and replicated by LIG3 activity, but if the gene contains mutations, enzymes necessary for energy production are not produced, potentially leading to central nervous system symptoms and muscle defects. © Fujita Health University DNA ligase…..
Trend Chart on Orphans - February 15th, 2020 FEATURE STORY ● The potential of gene therapy for mucopolysaccharidosis type I GENE THERAPY ● Repair of retinal degeneration following ex vivo Minicircle DNA gene therapy and transplantation of corrected photoreceptor progenitors RARE DISEASES ● Genetics and other omics in pediatric pulmonary…..
