Overall, our data provide evidence of successful therapeutic gene editing with TALE nucleases, into the mouse Mbs85 orthologous locus in fibroblasts and HPCs of a mouse model of FA-A, and establishes the rationale and the proof of principle to use this locus for the gene correction in other diseases to investigate the efficacy and safety of future gene editing strategies. This evidence concerns the gene PPP1R12C and Fanconi anemia complementation group A.