PPP1R12C and Fanconi anemia complementation group A: Our study demonstrates the feasibility of conducting a targeted gene therapy approach in embryonic fibroblasts and hematopoietic progenitors from a mouse model of FA-A using TALE nucleases as a gene editing platform, and highlights the potential of using for the first time the Mbs85 locus as a murine safe harbor for targeted integration, opening a new platform for in vitro and in vivo gene therapy applications in different disease models.