Schematic of various gene therapy strategies to treat β-thalassemia. (A) The use of lentiviral vectors to incorporate a full-length HBB gene or shmiRs targeting BCL11A for the upregulation of HbF. The use of CRISPR/Cas9 machinery can either knock out BCL11A to upregulate HbF (B) or incorporate a corrected HBB cassette with HDR machinery (C). Finally, adenine base editors can be used to convert A to G nucleotides to correct single-point mutations or incorrect splice sites, such as IVS1-110 (D). Created with BioRender.com.