Despite these difficulties, the recent announcement of the curative response of the first three patients (carrying a transfusion-dependent β-thalassemia and the SCD mutation) with CRISPR-Cas9-edited cells targeting BCL11A (CRISPR Therapeutics and Vertex CTX001 clinical trial2&3), clearly highlights the clinical potential of gene therapy. Here, BCL11A is linked to Schnyder corneal dystrophy.