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Gene Therapy

Blood Disorders Rare Diseases Respiratory Medicine Cerebral Adrenoleukodystrophy RNA Drugs Hematopoietic Stem Cells Heart Failure RNA-based Medicines Respiratory Gene Therapy In Vivo 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

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

Penn Researchers Develop Gene Therapy Tools for Advanced Retinal Degenerations

Gene Therapy Restores Immune Function in Children with Rare Disorder

Gene Therapy Reverses Heart Failure in Groundbreaking Animal Study