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Gene Therapy

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CRISPR Technique Reawakens Dormant Genes to Treat Blood Disorders

Early tests in human blood stem cells demonstrate the method’s potential to restore hemoglobin production with fewer risks than existing therapies

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

Study uncovers early-life window for in vivo gene therapy of blood stem cells

Researchers Release AI-Driven Tool for Custom CRISPR-Cas9 Enzyme Design

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