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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

First In Vivo CRISPR Gene Therapy Saves Newborn with Rare Genetic Disorder

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

New Immune Mechanism Linked to Duodenal Cancer Risk in FAP Patients

Colin Farrell and Kim Bordenave Seek Long-Term Care Facility for Son with Angelman Syndrome

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

Human-Specific Protein Variant Linked to Evolution of Language

Jeffrey Randall Allen Wins $10 Million on MrBeast's 'Beast Games'

Spinal Implant Shows Promise in Improving Muscle Function for SMA Patients