Patients affected by TDT and SCD, respectively, received a single intravenous infusion of CTX001 (autologous CRISPR-Cas9– edited CD34+ HSPCs) and monitored for engraftment, adverse events, total hemoglobin, and hemoglobin fractions on high-performance liquid chromatography [62]. This evidence concerns the gene CD34 and Schnyder corneal dystrophy.