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J., Daniel, S., Agid, Y., Javoy-Agid, F., Jenner, P., & Marsden, C

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A CRISPR/Cas9-mediated knockout of the GLA gene together with the overexpression of a number of different mutant variants allowed establishing a correlation between enzyme activity levels and ER retention [54] and, thereby, furthered the understanding of the full clinical presentation of Fabry disease

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