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Gene Therapy

Hearing Improvement Rare Diseases Respiratory Medicine Cerebral Adrenoleukodystrophy RNA Drugs Hematopoietic Stem Cells Heart Failure RNA-based Medicines Respiratory Gene Therapy In Vivo Gene Therapy Inherited Retinal Degenerations Metachromatic Leukodystrophy Lentiviral Vectors Severe Leukocyte Adhesion Deficiency Gene Regulation Clinical Trials Personalized Medicine RNA Interference Hearing Impairment MET Inhibitors Fabry Disease Genetic Deafness Vision Loss Treatment Duchenne Muscular Dystrophy Retinal Diseases Usher Syndrome Type 1B Blood Disorders Adeno-Associated Virus (AAV)

Kidney Organoids Reveal NFκB‑Linked AAV2 Toxicity, With Bardoxolone Methyl Mitigating Damage

The findings point to a human‑cell safety screen to catch risks before gene therapy reaches patients.

Dual-Vector Gene Therapy Restores Sight in Usher Syndrome Patient; Trial Advances to Higher Doses

Experimental Fabry Gene Therapy Achieves Near-Break-Even Costs and Advances to Larger Study

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

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