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Gene-replacement therapies are transforming children’s lives

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Several therapies to correct severe genetic disorders have been approved by medical regulators in 2022, and others have produced impressive clinical trial results

Health



23 December 2022

DNA

Gene-replacement therapies involve replacing DNA that doesn’t work properly

Shutterstock/SWKStock

This year has been full of good news stories about gene-replacement therapies alleviating severe genetic disorders in children, suggesting they are finally living up to their promise after a bumpy few decades.

“We’re curing kids of fatal diseases. It’s wonderful,” Donald Kohn at the University of California, Los Angeles told New Scientist in May after successfully using a gene-replacement therapy to reverse a rare life-threatening immune condition called leukocyte adhesion deficiency in nine children.

Gene-replacement therapies are used to correct …

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