Abstract
Gene therapy for hematopoietic stem cells （HSCs） holds promise as a treatment for various genetic diseases. HSCs constitute peripheral blood throughout life, and thus, repairing pathogenic mutations and deletions in HSCs can allow for the lifelong treatment of genetic diseases. Autologous HSC gene therapy has been developed through lentiviral gene addition and gene editing in patient HSCs, and no requirement of a compatible donor makes this therapy applicable to most patients. The efficacy of HSC gene therapy using lentiviral vectors and gene editing has been proven in recent clinical trials; however, the complexity and high cost due to ex vivo culture have hindered widespread use. Therefore, the development of in vivo HSC gene therapy, which can directly deliver gene therapy tools to bone marrow HSCs by systemic administration, is a desirable solution.
