Time-resolved functional analysis of acute impairment of frataxin expression in an inducible cell model of Friedreich ataxia

Dörte Poburski, Josefine Barbara Boerner, Michel Koenig, Michael Ristow, René Thierbach. Biology Open 2016 : doi: 10.1242/bio.017004

OPEN ACCESS

Here, we have developed a new mammalian cell model employing the Cre/loxP recombination system to induce a homozygous or heterozygous frataxin knockout in mouse embryonic fibroblasts. Induction of Cre-mediated disruption by tamoxifen was successfully tested on RNA and protein levels. The robustness of the newly established system may additionally be used for a time-resolved study of pharmacological candidates in a HTS manner.

 Time-resolved functional analysis of acute impairment of frataxin expression in an inducible cell model of Friedreich ataxia