Humanized mouse models have been developed to study human hematopoiesis and therapeutic application of hematopoietic stem cell transplantation. To evaluate the safety and efficacy of lentiviral vectors for gene therapy, human CD34+ cells have been transduced with lentiviral vectors and transplanted into the humanized mice. Recipient mice are monitored over time and sacrificed for bone marrow analyses with regard to human cell engraftment, lineage distribution, and vector transduction. This chapter details the procedure for lentiviral transduction and transplantation of human hematopoietic stem/progenitor cells into humanized mice to study inherited human hematological disorders.
Keywords: Engraftment; Hematopoietic stem cells (HSC); Humanized mouse models; Lentiviral vector; Transduction; Transplantation.