Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis

Abstract

Gene therapy using autologous or allogeneic cells offers promising possibilities to treat inherited and acquired diseases, ideally leading to a long-lasting therapeutic correction. This article summarizes efforts that use integrating vectors derived from retroviruses and transposons, and briefly explains integrating vector biology and integration site analysis and recent successful application of this technology in clinical trials. Moreover, outlined is how these vectors can be used for cancer gene discovery and clonal tracking of benign and malignant hematopoiesis to gain insights into the dynamics of hematopoiesis.

Keywords: Clonal tracking; Gene therapy; Hematopoietic dynamics; Insertion site analysis; Lentiviral vector; Retroviral vector; Stem cells; Transposon.