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Gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions.

KJ Muldoon of Clifton Heights, Pa. was born with severe CPS1 deficiency, a rare and dangerous genetic disease estimated by some experts to affect around one in a million babies. Those infants lack an enzyme needed to help remove ammonia from the body, so it can build up in their blood and become toxic. Baby KJ is growing and thriving after getting an experimental gene editing treatment made just for him by a team at Children’s Hospital of Philadelphia and Penn Medicine.
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