Correction of the frataxin gene using CRISPR

Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich Ataxia. D L Ouellet, K Cherif, J Rousseau and J-P Tremblay; Gene Therapy, accepted article preview 26 December 2016; doi: 10.1038/gt.2016.89.

Short title: Correction of the frataxin gene using CRISPR

Here, we use the CRISPR-Cas9 system to remove the mutated GAA expansion and restore the frataxin gene transcriptional activity and protein level. Both YG8R and YG8sR mouse models and cell lines derived from these mice were used to CRISPR-edited successfully the GAA expansion in vitro and in vivo.
All experiments present in this article help to identify the most suitable mouse model for gene editing in vivo, hoping this will quickly lead to human clinical trials in Friedreich ataxia patients. However, as any kind of therapy, the side effects of a treatment must be clearly defined and known before going further with the massive treatment of patients.

Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich Ataxia.