A random, short-hairpin-RNA (shRNA)-expressing library was screened in primary FRDA fibroblasts and identified two shRNAs that reverse the growth/viability defect in an in vitro model of FA. One of these two clones increases frataxin expression in primary FRDA fibroblasts, either as a vector-expressed shRNA or as a transfected short-interfering RNA (siRNA).

Read More: Phenotypic Screening for Friedreich Ataxia Using Random shRNA Selection