Several therapies to right extreme genetic problems have been authorised by medical regulators in 2022, and others have produced spectacular scientific trial outcomes
Health
23 December 2022
Gene-replacement therapies contain changing DNA that doesn’t work correctly Shutterstock/SWKStock
This yr has been full of excellent news tales about gene-replacement therapies assuaging extreme genetic problems in youngsters, suggesting they’re lastly dwelling as much as their promise after a bumpy few a long time.
“We’re curing kids of fatal diseases. It’s wonderful,” Donald Kohn on the University of California, Los Angeles advised New Scientist in May after efficiently utilizing a gene-replacement remedy to reverse a uncommon life-threatening immune situation referred to as leukocyte adhesion deficiency in 9 youngsters.
Gene-replacement therapies are used to right …