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Showing 1 to 1 of 1 for “"spinal cord organoid"”.

  1. MODELLING RIBOFLAVIN TRANSPORTER DEFICIENCY (RTD) USING IPSC-DERIVED MODELS TO TEST GENE THERAPY EFFICACY

    … three disease models through 2D motoneurons, spinal cord and retinal organoid differentiation of RTD patients-specific induced pluripotent (iPSC) line carrying several mutations in SLC52A2 gene. First, we tested gene replacement therapy on RTD type 2 patient-derived motoneurons using an …

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