Adeno-Associated Virus OTOF Gene Sickle Cell Disease Gene Delivery Systems Clinical Applications Clinical Trials CRISPR Technology CCR5 Mutation Advances in Gene Therapy KCNJ2 as a Protective Factor Stem Cell Research Klotho Protein CPS1 Deficiency AAV Vectors Neurodegenerative Diseases Deafness Duchenne Muscular Dystrophy OTOF Gene Therapy Spinal Muscular Atrophy Ophthalmology Usher Syndrome Lentiviral Vectors Craniosynostosis Rare Diseases Viral Vectors Adeno-Associated Viruses Cardiomyocytes Cardiac Function Photoreceptor Function
Phase 1/2 safety trials in human newborns are being planned after primate studies showed durable HIV protection, with researchers addressing immunogenicity, strain diversity, production scalability.