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Gene therapy for mitochondrial disease shows promise in animals By (0) Results from a long-term study of a gene therapy technique to prevent inherited mitochondrial disease show promise, researchers say. Studies of the technique at Oregon Health & Science University in Portland show no adverse health effects in rhesus macaque monkeys and their offspring. The researchers said the technique could break the cycle of disease passed from mother to baby through mutations in mitochondrial DNA, or mtDNA. Advertisement Though results of animal studies sometimes differ in humans, the findings bolster the scientific basis for mitochondrial replacement therapy, or MRT, in human clinical trials, the study authors said.

United statesUnited kingdomShoukhrat mitalipovPaula amatoUs congressUnited mitochondrial disease foundationSchool of medicineOregon national primate research centerOregon health science university in portlandNational human genome research instituteDrug administrationOregon healthScience universityUnited mitochondrial diseaseNational primate research centerEmbryonic cell