Gene therapy applications to transfusion medicine

This chapter focuses on the possible applications of gene therapy to transfusion medicine. Gene therapy falls within the purview of transfusion medicine when it requires the infusion of either gene-modified cells or direct administration of vectors that contain the therapeutic genetic material. The chapter summarizes the salient features of the viral vectors. It discusses nonviral techniques that are under development for gene therapy. Replication-competent viruses develop either by recombination of the constituent parts of the vector system with endogenous viral-sequences in the vector packaging cell lines or by activation of endogenous proviral-sequences. The chapter also discusses three types of genetic diseases such as hemophilia B, hemophilia A and hemoglobinopathies, where hemophilia B and hemophilia A are caused by a deficiency in factor IX and factor VIII respectively. It further describes gene-editing approaches such as transcription activator-like effector nucleases, zinc finger nucleases, and clustered regularly interspaced short palindromic repeats.