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Gene Therapy

Clinical Trials Innovative Treatments Public Health Innovations Genetic Disorders Hemophilia Treatment CRISPR Technology RNA Interference Patient Safety Treatment Innovations Duchenne Muscular Dystrophy Fetal Hemoglobin Research

Preclinical mRNA Platform Shows Broad-Spectrum Antiviral Protection in Rodents

The intranasal lipid-nanoparticle system transiently expresses ten ISG proteins to block diverse viruses for up to four days.

Vinay Prasad Resigns as FDA Biologics Director After Politicized Tenure

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

Pfizer Withdraws Sickle Cell Drug Oxbryta Over Safety Concerns

Breakthrough Gene Therapies Offer New Hope for Sickle Cell Disease