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A research team led by Prof. Li Yunhai from the Institute of Genetics and Developmental Biology (IGDB) of the Chinese Academy ...
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ParentingPatch on MSNInfant With Incurable Disease Becomes First Personalized Gene Therapy SuccessInfant KJ Muldoon was born with carbamoyl phosphate synthetase 1 (CPS1) deficiency, a condition so rare that only about 1 in ...
After 3 doses of tailor-made treatment, an infant with a rare, life-threatening urea cycle disorder is thriving ...
A baby known as KJ is the first person in the world to receive a customized CRISPR therapy designed to fix a specific ...
A tailored CRISPR base-editing therapy was given for the first time to an infant who was born with a rare genetic disease, with promising effects, researchers reported.
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