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www.mext.go.jp/kaigisiryo/2019/09/__icsFiles/afieldfile/2019/09/11/1421121_002.pdf
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www.jbc.org/article/S0021-9258(19)81813-6/fulltext
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761052Orig1s000SumR.pdf
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www.nature.com/articles/s41587-025-02947-8
Dec 20, 2025
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www.nature.com/articles/s41598-018-22456-w
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Open Targets Platform | Open Targets Platform Documentation
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Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models
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