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The route of administration was noted to affect quality of life as was frequency of dosing, although the magnitude of impact in the economic evaluation was considered large based on clinical expert opinion

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AG-haESC culture and transfection AG-haESCs carrying deletions in the H19 -DMR and IG -DMR regions, as well as an Oct4-EGFP transgene (termed H19 DMR - IG DMR -AGH-OG3 or 408), were used to generate EFS-C9 and CAG-C9 cells for genetic screening