Future perspectives on hematopoietic stem cell gene therapy

Abstract

Hematopoietic stem cells have the ability to self-renew and differentiate into multilineage cells. Therefore, hematopoietic stem cell gene therapy, which involves introducing therapeutic genes into these cells, could be an effective treatment for hereditary diseases currently targeted by hematopoietic cell transplantation. In fact, gene therapies using lentiviral vectors have already received manufacturing and sales approvals for several hereditary diseases. However, there are issues with this approach, such as tumorigenesis associated with the insertion of the vector genome and insufficient response to gain-of-function diseases caused by proteins derived from mutant genes. For this reason, the development of gene therapy using genome editing technology has become an active area of research in recent years. Nevertheless, because these technologies may cause permanent changes to the human genome, it is essential to proceed carefully with clinical development, based on social consensus.