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Gene Therapy

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Sarepta Halts Elevidys Shipments for Non-Ambulatory Patients After Second Fatal Liver Failure

Sarepta is enlisting experts to design a stronger immunosuppressive regimen.

Gene Therapy Disables HSV-1’s Uracil-DNA Glycosylase and Protects against Encephalitis

First Personalized CRISPR Therapy Successfully Treats Infant’s Rare Genetic Disorder

Gene Therapy Restores Immune Function in Children with Rare Disorder

First Death Reported from Sarepta's Duchenne Gene Therapy Raises Safety Concerns

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

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