The efficacy and specificity of ZFNs designed for the targeting of the AAVS1 “safe harbor” locus, together with the high number of private mutations characterized in FA‐A patients, suggest that the specific insertion of FANCA in the AAVS1 site of FA‐A patient's HSPCs would result in a particularly useful platform for restoring normal hematopoiesis in these patients. The gene discussed is PPP1R12C; the disease is Fanconi anemia complementation group A.