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Gene Therapy

Gene Therapy Medical Treatments Genetic Disorders Clinical Trials Patient Care Gene Editing Treatment Options Regulatory Approval Pediatric Care Reproductive Rights

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.

Gene Therapy Restores Immune Function in Children with Rare Disorder

NHS Approves First Gene-Editing Therapy for Severe Sickle Cell Disease