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Gene Therapy

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

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.