Modelling precise responses to anti-seizure medication using brain organoids carrying SCN2A-GoF and SCN2A-LoF mutations

Yuling Yang , Yi Yan , Yang Cai , Xin Wang , Zhicheng Shao , Jing Ding

Clinical and Translational Medicine ›› 2026, Vol. 16 ›› Issue (4) : e70666

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Clinical and Translational Medicine ›› 2026, Vol. 16 ›› Issue (4) :e70666 DOI: 10.1002/ctm2.70666
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Modelling precise responses to anti-seizure medication using brain organoids carrying SCN2A-GoF and SCN2A-LoF mutations
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Yuling Yang, Yi Yan, Yang Cai, Xin Wang, Zhicheng Shao, Jing Ding. Modelling precise responses to anti-seizure medication using brain organoids carrying SCN2A-GoF and SCN2A-LoF mutations. Clinical and Translational Medicine, 2026, 16 (4) : e70666 DOI:10.1002/ctm2.70666

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Yang Y, Cai Y, Wang S, et al. Human cortical organoids with a novel SCN2A variant exhibit hyperexcitability and differential responses to anti-seizure compounds. Neurosci Bull. 2025; 41(11): 2010–2024.

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Amador A, Bostick CD, Olson H, et al. Modelling and treating GRIN2A developmental and epileptic encephalopathy in mice. Brain. 2020; 143(7): 2039-2057.

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Jia L, Li M, Pachernegg S, et al. Variant-specific in vitro neuronal network phenotypes and drug sensitivity in SCN2A developmental and epileptic encephalopathy. J Neurochem. 2024; 168(12): 3950-3961.

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2026 The Author(s). Clinical and Translational Medicine published by John Wiley & Sons Australia, Ltd on behalf of Shanghai Institute of Clinical Bioinformatics.

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