What happened
A team of doctors and scientists has used a tailor-made gene-editing therapy to treat an infant with a rare genetic condition, a medical first that opens the door to a new era of individualized medicine, especially for people with uncommon diseases, the researchers reported Thursday in The New England Journal of Medicine. "This is the future of medicine," said study coauthor Dr. Kiran Musunuru, a gene-editing expert at the University of Pennsylvania.
Who said what
KJ Muldoon was diagnosed with CPS1 deficiency, which causes toxic levels of ammonia in the blood, soon after his premature birth in Philadelphia last August. He had "perhaps as few as six months before a mounting risk of severe brain damage or death," The New York Times said. Instead, he "made medical history."
KJ's diagnosis launched an "all-out six-month sprint" by a network of researchers and companies to develop a therapy, "vet its safety and use it to fix an errant gene" in his liver, The Washington Post said. They used the gene-editing tool CRISPR to create an enzyme that "flips the mutated DNA 'letter'" to the "correct type" to process ammonia, The Associated Press said. KJ got his first injection in February, then lower doses in March and April.
The Week
Escape your echo chamber. Get the facts behind the news, plus analysis from multiple perspectives.
Sign up for The Week's Free Newsletters
From our morning news briefing to a weekly Good News Newsletter, get the best of The Week delivered directly to your inbox.
From our morning news briefing to a weekly Good News Newsletter, get the best of The Week delivered directly to your inbox.
What next?
"We're still very much in the early stages of understanding what this medication may have done for KJ," said Dr. Rebecca Ahrens-Nicklas, his doctor at Children's Hospital of Philadelphia and a study coauthor. "But every day, he's showing us signs that he's growing and thriving." He is expected to go home in a few weeks.
-
The 7 best police procedurals of all timeThe Week Recommends There’s more to cops and robbers than just nabbing the bad guy at the end of the show
-
Shutdown stalemate nears key pain pointsSpeed Read A federal employee union called for the Democrats to to stand down four weeks into the government standoff
-
Navy jet, helicopter crash half-hour apart off carrierSpeed Read A US Navy helicopter and a fighter jet both crashed in the same half-hour during separate operations
-
Dinosaurs were thriving before asteroid, study findsSpeed Read The dinosaurs would not have gone extinct if not for the asteroid
-
SpaceX breaks Starship losing streak in 10th testspeed read The Starship rocket's test flight was largely successful, deploying eight dummy satellites during its hour in space
-
Rabbits with 'horns' sighted across Coloradospeed read These creatures are infected with the 'mostly harmless' Shope papilloma virus
-
Lithium shows promise in Alzheimer's studySpeed Read Potential new treatments could use small amounts of the common metal
-
Scientists discover cause of massive sea star die-offSpeed Read A bacteria related to cholera has been found responsible for the deaths of more than 5 billion sea stars
-
'Thriving' ecosystem found 30,000 feet underseaSpeed Read Researchers discovered communities of creatures living in frigid, pitch-black waters under high pressure
-
How mice with two dads bring us closer to two men having a child of their ownThe Explainer Science breakthrough produces healthy, fertile mice from two sperm cells and an empty egg
-
Bacteria can turn plastic waste into a painkillerUnder the radar The process could be a solution to plastic pollution
