Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases.

We examined the efficiency, specificity, and off-target mutation rates of three different genome editing tools: zinc finger nucleases (ZFNs), transcriptional activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 systems. Our results indicated that the ZFNs were best at making the targeted gene editing mutation, and had the least off-target mutations generated compared to TALENs and CRISPER/Cas9 systems.