AP Spotlight Gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions LAURA UNGAR Associated Press May 16, 2025 May 16, 2025 Updated Nov 19, 2025 0 Facebook Twitter Bluesky WhatsApp SMS Email Print Copy article link Save A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him.kAm#6D62C496CD 56D4C:365 E96 42D6 :? 2 ?6H DEF5J[ D2J:?8 96VD 2>@?8 E96 7:CDE E@ 36 DF446DD7F==J EC62E65 H:E9 2 4FDE@> E96C2AJ E92E D66<D E@ 7:I 2 E:?J 3FE 4C:E:42= 6CC@C :? 9:D 86?6E:4 4@56 E92E <:==D 92=7 @7 27764E65 :?72?ED]k^AmkAm%9@F89 :E >2J 36 2 H9:=6 367@C6 D:>:=2C A6CD@?2=:K65 EC62E>6?ED 2C6 2G2:=23=6 7@C @E96CD[ 5@4E@CD 9@A6 E96 E649?@=@8J 42? 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Exercise boosts survival rates in colon cancer patients, study shows A first-of-its-kind experiment shows a three-year exercise program improved survival in colon cancer patients.Â
US organ donation system faces scrutiny and changes after reports of disturbing near-misses The U.S. is developing new safeguards for the organ transplant system after an investigation found a Kentucky group continued preparations for…
Snakes have bitten this man hundreds of times. His blood could help make a better treatment A Wisconsin man has been bitten by snakes hundreds of times, and scientists are studying his blood to treat snakebite.
Cancer before age 50 is increasing. A new study looks at which types The government study provides the most complete picture yet of early-onset cancers, finding the largest increases in four types.
Whooping cough cases are rising again in the US Preliminary data from the CDC says the U.S. has seen 8,485 cases of whooping cough in 2025. That's twice as many cases as this time last year.