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 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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Sign up! * I understand and agree that registration on or use of this site constitutes agreement to its user agreement and privacy policy. Related to this story 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
Science Snakes have bitten this man hundreds of times. His blood could help make a better treatment ADITHI RAMAKRISHNAN AP Science Writer
Wellness Cancer before age 50 is increasing. A new study looks at which types CARLA K. JOHNSON Associated Press
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