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Gene Therapy

Synovial Sarcoma Transfusion-Dependent Beta Thalassemia Sickle Cell Disease Cerebral Adrenoleukodystrophy (CALD) CAR T-cell Therapy AADC Deficiency Childhood Blindness

First Death Reported in Patient Using Sarepta's Duchenne Gene Therapy

Gene Therapy Restores Sight in Children Born Blind Due to Rare Eye Disorder

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

Groundbreaking Lupus Treatment Trial Offers Hope for Drug-Free Remission

NHS Approves Groundbreaking Gene-Editing Therapy for Beta Thalassemia

FDA Approves Adaptimmune's Groundbreaking T-Cell Therapy for Rare Cancer