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Gene replacement therapies are changing children's lives

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

Health


December 23, 2022

DNA

Gene replacement therapies replace DNA that is not working properly

Shutterstock/SWKStock

This year has been full of good news stories about gene replacement therapies alleviating serious genetic disorders in children, suggesting that after a few bumpy decades, they are finally delivering on their promise.

“We cure children of deadly diseases. It’s amazing,” said Donald Kohn of the University of California, Los Angeles New scientist in May after successfully applying a gene replacement therapy to reverse a rare life-threatening immune disorder called leukocyte adhesion deficiency in nine children.

Gene replacement therapies are used to…

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