The present invention relates to a method for genetically editing an enhancer site of hematopoietic stem cell BCL11A efficiently and safely. In the present invention, it is confirmed that hematopoietic stem cells which have been modified have normal cell functions and increase the expression of the fetal hemoglobin significantly to counteract the normal-hemoglobin deletion in thalassemia patients. Therefore, genetically edited hematopoietic stem cells can be used for clinical treatment of β-thalassemia and sickle cell anemia.本發明關於高效安全基因編輯造血幹細胞BCL11A的增強子位點的方法。在本發明中，已確認能夠修飾完成後的造血幹細胞具有正常細胞功能，且顯著提高胎兒血紅蛋白的表達, 以抵消地中海貧血患者缺失的正常血紅蛋白。因此，可將基因編輯後的造血幹細胞用於β-地中海貧血和鐮刀紅細胞貧血的臨床治療。
