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Effect on protein expression of adding dsRNA siGAA (25 nM) to homozygous FA patient-derived cell lines or a cell line derived from a heterozygous donor. (a) Western analysis of FXN protein expression in different FRDA cell lines after transfection with siGAA. (b) Quantification of data shown in (a), (n = 3). CM is a noncomplementary negative control RNA. Numbers of GAA repeats in two alleles of patient FXN genes are shown in parentheses.
