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Australia has become the second country in the world to explicitly authorize and regulate mitochondrial replacement therapies, offering hope to some families who want to have children but fear passing along inherited mitochondrial diseases to them.
These diseases affect the mitochondria, tiny structures in nearly every cell that generate the energy it needs to survive and function and, unlike cellular components other than the nucleus, have their own DNA. Underperforming mitochondria can lead to problems in organs like the heart, lungs, or eyes, and symptoms may progress and expand to other organs over time.
In the U.S., up to 4,000 children are born each year with a mitochondrial disease. Some die early; others cope with potentially painful or debilitating symptoms with treatments like physical therapy and medications. When mitochondrial diseases result from mutations in the mitochondrial DNA, rather than in the nuclear genome, the way mitochondria are inherited can cause problems in children even when their parents have no or mild symptoms.
Mitochondrial replacement does what the name suggests: it swaps out diseased mitochondria that a biological mother would pass...