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Gene Therapy

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

Six-month outcomes reveal an average hearing improvement from 106 to 52 decibels across all age groups under a safe, well-tolerated protocol.

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