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Gene Therapy

Gene Therapy Restores Hearing in Most With OTOF Deafness, With Gains Lasting 2.5 Years

The multicenter trial shows the strongest gains in young children, signaling a path to clinical review in China.

One-Time CRISPR Edit Halves LDL and Triglycerides in Early Human Trial

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

CRISPR Technique Reawakens Dormant Genes to Treat Blood Disorders

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

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

Philadelphia Doctors Pioneer Personalized Gene Therapy for Rare Metabolic Disorder