Gene-replacement therapies are transforming children’s lives

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

By Alice Klein

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 …