Adeno-Associated Virus Clinical Trials CRISPR Technology Gene Delivery Systems Clinical Applications Clinical Research Sickle Cell Disease OTOF Gene Advances in Gene Therapy KCNJ2 as a Protective Factor Stem Cell Research Klotho Protein CPS1 Deficiency AAV Vectors Neurodegenerative Diseases RNA Technology Deafness Duchenne Muscular Dystrophy OTOF Gene Therapy Spinal Muscular Atrophy Ophthalmology Usher Syndrome Therapeutic Applications Haploinsufficiency Huntington's Disease Craniosynostosis CAR-T Therapy Kallikrein Inhibition SYNGAP1-related Disorders Adenosine Deaminase Deficiency Cyclin A2 CRISPR-Cas9 Personalized Medicine Genome Editing Enzyme Deficiency SMN1 Gene Lentiviral Vectors Stem Cell Treatment Rare Diseases Viral Vectors Adeno-Associated Viruses Cardiomyocytes Cardiac Function Photoreceptor Function CCR5 Mutation
Regulators based the decision on 52-week motor gains with a consistent safety profile.
