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Gene Therapy

Adeno-Associated Virus Clinical Trials Clinical Research Sickle Cell Disease CRISPR Technology Gene Delivery Systems OTOF Gene Clinical Applications CAR-T Therapy Advances in Gene Therapy KCNJ2 as a Protective Factor Stem Cell Research Klotho Protein Kallikrein Inhibition SYNGAP1-related Disorders CPS1 Deficiency AAV Vectors Neurodegenerative Diseases RNA Technology Adenosine Deaminase Deficiency Deafness Duchenne Muscular Dystrophy OTOF Gene Therapy Spinal Muscular Atrophy Ophthalmology Usher Syndrome Viral Vectors Rare Diseases Lentiviral Vectors Therapeutic Applications Haploinsufficiency Huntington's Disease Craniosynostosis Adeno-Associated Viruses Cardiomyocytes Cardiac Function Photoreceptor Function CCR5 Mutation