FIG. 2.

Effect of adding dsRNA siGAA to homozygous FA patient-derived cell lines or a cell line derived from a heterozygous donor. (a) Sequences of siGAA or noncomplementary dsRNA control CM. (b) Relative expression of FXN mRNA evaluated by qPCR in different FRDA cell lines. (c) Effect of transfecting siGAA (25 nM) on FXN expression (n = 3) in varied cell lines. CM is a noncomplementary negative control RNA. Numbers of GAA repeats in two alleles of patient FXN genes are shown in parentheses. dsRNA, duplex RNA; FA, Friedreich's ataxia; qPCR, quantitative polymerase chain reaction.