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Proposed Design for a Natural History Study of SCN1A-Positive (SCN1A+) Dravet Syndrome
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Efficient Delivery of Clinically-Validated Adeno-Associated Viral (AAV) Vectors to the Central Nervous System: A Systematic Evaluation of Multiple Routes of Administration and Viral Serotypes in Cynomolgus Macaques
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From Gene Replacement to Gene Regulation: Developing a Disease-Modifying AAV Gene Therapy Vector for SCN1A-Positive (SCN1A+) Dravet Syndrome