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Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonhuman Primates.
Hum Gene Ther
; 33(11-12): 579-597, 2022 06.
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| MEDLINE | ID: mdl-35435735
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