Dissecting the Lives of Hematopietic Cells at a Clonal Level: Appropriate Models and Implications for Gene Therapies Aging and HSC Disorders

Blood-forming stem cells replenish the blood and immune system, making them important targets for gene therapy and transplantation. Cynthia Dunbar, M.D., explains how genetic barcodes let scientists track individual stem cell clones over time. Long-term studies in nonhuman primates reveal stable contributions from blood stem cells and unexpected persistence among certain natural killer immune cells. Dunbar reviews successes and safety concerns in gene therapy, including leukemias associated with some integrating viral vectors. She compares these approaches with CRISPR gene editing, examining challenges in precise gene insertion, stem cell recovery after transplantation, and age-related clonal hematopoiesis. She also addresses treatment costs, intensive preparation for transplantation, and barriers to patient access. These insights help researchers evaluate new treatments more carefully and work toward safer, more accessible care. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40858]