At present, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the radical treatment of most hereditary hematological diseases and primary immunodeficiency diseases. However, due to the lack of human leukocyte antigen (HLA) compatible donors and the possibility of graft-versus-host disease (GVHD) after allo-HSCT, the general application of this method is limited. In recent years, gene-editing techniques and stem cell biotechnology have developed rapidly, and gene therapy using patients′ autologous hematopoietic stem/progenitor cells (HS/PC) has become increasingly popular, which has become a focus for research aera of gene therapy for hereditary hematological diseases. On the one hand, this approach solves the problem of immune rejection. On the other hand, the discovery of the clustered regularly interspaced short palindromic repeat sequences (CRISPR)- CRISPR-associated protein (Cas)9 system makes targeted gene therapy more feasible. CRISPR-Cas9 system can shear targeted genes at a specific point, so as to achieve the purpose of genomic DNA modification. Therefore, this review mainly focuses on gene-editing techniques, clinical research progress of gene therapy in hereditary hematological diseases, the challenges and future development of gene therapy.
