Gene-replacement therapies are transforming children’s lives

A number of therapies to appropriate extreme genetic problems have been authorized by medical regulators in 2022, and others have produced spectacular scientific trial outcomes

By Alice Klein

This yr has been full of fine information tales about gene-replacement therapies assuaging extreme genetic problems in kids, suggesting they’re lastly dwelling as much as their promise after a bumpy few many years.

“We’re curing youngsters of deadly illnesses. It’s fantastic,” Donald Kohn on the College of California, Los Angeles advised New Scientist in Could after efficiently utilizing a gene-replacement remedy to reverse a uncommon life-threatening immune situation known as leukocyte adhesion deficiency in 9 kids.

Gene-replacement therapies are used to appropriate …