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Gene Therapy

CRISPR Technology Zolgensma Genetic Disorders CAR T-cell Therapy CRISPR Medical Innovations Patient Care Cost of Treatment Neuromuscular Disorders Congenital Deafness Clinical Trials

Personalized CRISPR Therapy Sends Baby KJ Home from CHOP

FDA fast-tracked the six-month custom CRISPR therapy that corrected KJ’s CPS1 mutation, enabling normal protein metabolism with reduced drug support.