Genetic Engineering Publications - GEG Tech top picks

Friedreich’s ataxia (FRDA) is an autosomal recessive neurological disease caused by expansions of GAA repeats in intron 1 of the frataxin (FXN) gene. The authors generated zinc finger nuclease (ZFN) to excise the expanded GAA repeats. This correction persisted during reprogramming of ZFN-edited fibroblasts to induced pluripotent stem cells (iPSCs) and subsequent differentiation into neurons. They observed that he expression of FRDA biomarkers were reversed in ZFN-corrected neuronal cells.

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