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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 Sickle Cell Disease Usher Syndrome Type 1B Adeno-Associated Virus (AAV) Immunotherapy Huntington's Disease Neurological Disorders Hearing Loss Blood Disorders ADA-SCID

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

New long-term results validate a path to broader access through cryopreserved manufacture.