The growth of human hematopoietic cells in immune-deficient mice promises to revolutionize our ability to study the normal developmental program of human hematopoiesis and the biological consequences of aberrant proliferation and differentiation. Advances in stem cell purification will require assays to test for function, and the identification and the characterization of novel hematopoietic growth factors will be aided by in vivo experiments. The engraftment of hematopoietic cells directly from patients with disease should ultimately lead to animal models for many human hemopathies and leukemias. Already important preliminary experiments have established the feasibility of such models for leukemia, cancer, infectious diseases, and autoimmunity. The production of human antibodies directed against toxic agents for which humans cannot be immunized could provide the basis for improved pharmaceuticals. Although an important foundation has been laid, much work remains to explore the full potential of this mouse transplantation system.