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Employing CRISPR/Cas9, we first generated a complete gbh-1 knockout model, gbh-1 (ko), and then introduced variants equivalent to those identified in our patients: gbh-1(D72G) for p.Asp59Gly, gbh-1(G283R) for p.Gly263Arg, and gbh-1(G247Vfs6) for p.Gly227Valfs*6 (Table 2 and Fig

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