Discussion on the Druggability of Hematopoietic Stem Cells

HSCs (hematopoietic stem cells) are a class of adult tissue stem cells with the capacity for self-renewal and multilineage differentiation. Since the first bone marrow transplantation in 1956 and the initial report of HSCs in 1961, both clinical practice and basic research on HSCs have advanced for more than six decades. As a form of personalized cellular therapy, HSC transplantation exerts dual effects of hematopoietic reconstitution and immune modulation, and has become one of the curative strategies for hematologic malignancies. With continuous progress in ex vivo expansion, gene editing, induced differentiation, and chimeric antigen receptor technologies, HSC-based gene therapy has emerged as an effective treatment approach for congenital genetic disorders and autoimmune diseases. The development and regulatory approval of several HSC-based products have driven the field from a personalized technology toward productization. This review summarizes the current applications and clinical outcomes of approved and investigational HSC-based products, highlights the key challenges in advancing HSCs toward drug development, and provides perspectives for further translational research, which may also serve as a reference and paradigm for the development and clinical application of other adult tissue stem cell-based therapies.

CSTR: 32200.14.cjcb.2026.01.0006