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Gene Therapy Shows No Long-Term Harm in Animals: Study

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 (mtDNA).

Though results of animal studies sometimes differ in humans, the findings bolster the scientific basis for mitochondrial replacement therapy (MRT) in human clinical trials, the study authors said.