Two Halves Make a Whole .
作者信息
Wong Jennifer C
机构信息
Departments of Human Genetics and Pediatrics, Emory University, Atlanta, GA, USA.
出版信息
Epilepsy Curr. 2025 Jun 30:15357597251355871. doi: 10.1177/15357597251355871.
Abstract
摘要
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本文引用的文献
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Antisense oligonucleotide treatment in a preterm infant with early-onset SCN2A developmental and epileptic encephalopathy.反义寡核苷酸治疗早发型SCN2A发育性和癫痫性脑病的早产儿
Nat Med. 2025 Apr 22. doi: 10.1038/s41591-025-03656-0.
2
Interneuron-specific dual-AAV gene replacement corrects epileptic phenotypes in mouse models of Dravet syndrome.中间神经元特异性双腺相关病毒基因替代可纠正Dravet综合征小鼠模型中的癫痫表型。
Sci Transl Med. 2025 Mar 19;17(790):eadn5603. doi: 10.1126/scitranslmed.adn5603.
3
Viral vector-mediated expression of NaV1.1, after seizure onset, reduces epilepsy in mice with Dravet syndrome.病毒载体介导的 NaV1.1 表达，在癫痫发作后，可减少 Dravet 综合征小鼠的癫痫发作。
J Clin Invest. 2023 Jun 15;133(12):e159316. doi: 10.1172/JCI159316.
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Cell-Selective Adeno-Associated Virus-Mediated Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonhuman Primates.细胞选择性腺相关病毒介导的基因调控治疗挽救 Dravet 综合征小鼠模型的死亡率和癫痫发作表型，并且在非人灵长类动物中具有良好的耐受性。
Hum Gene Ther. 2022 Jun;33(11-12):579-597. doi: 10.1089/hum.2022.037.
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Scn1a gene reactivation after symptom onset rescues pathological phenotypes in a mouse model of Dravet syndrome.症状出现后 Scn1a 基因再激活可挽救 Dravet 综合征小鼠模型的病理性表型。
Nat Commun. 2022 Jan 10;13(1):161. doi: 10.1038/s41467-021-27837-w.
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Transfer of to the brain of adolescent mouse model of Dravet syndrome improves epileptic, motor, and behavioral manifestations.将[具体物质未给出]转移至Dravet综合征青少年小鼠模型的大脑可改善癫痫、运动及行为表现。
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Sci Transl Med. 2020 Aug 26;12(558). doi: 10.1126/scitranslmed.aaz6100.
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J Neurosci. 2018 Sep 5;38(36):7912-7927. doi: 10.1523/JNEUROSCI.0193-18.2018. Epub 2018 Aug 13.
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Mapping genetic modifiers of survival in a mouse model of Dravet syndrome.绘制德拉韦特综合征小鼠模型中生存的遗传修饰因子图谱。
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