Advanced Applications of Gene Therapy in the Treatment of Haematologic Disorders

Gene therapy is an experimental technique for treating genetic disorders; it is designed to introduce a functional gene into a specified cell instead of missing or defective ones in order to correct the disease process by restoring, modifying or improving the cellular function. The past decade has been characterised by extreme advances in gene therapy which are considered an avenue of hope for a number of blood diseases that are difficult to be effectively treated with medications, recombinant therapeutic proteins or even transplantation. A number of preclinical and clinical studies have been conducted to develop gene therapy for patients with various genedeficient haematological disorders, i.e. haemophilia, β-haemoglobinopathies, leukaemia, lymphoma and other haematologic. Results from some clinical trials indicate that gene therapy can cure or improve many inherited or acquired hematologic disorders. It has the potential to improve the quality of life of those patients who are fully dependent on life-long parenteral therapy with recombinant proteins or blood and its products. However, the increased knowledge about the biological basis of the haematological disorders is associated with important advances in therapeutic management prevention of new cases. In this chapter, the current status of gene therapy will be reviewed with a focus on recent technologies such as gene delivery vectors, CRISPR genome editing technology, and CAR T-cell therapy, and their applications in the treatment of various blood disorders, in addition to the demonstrations of the outcomes of selected clinical studies, followed by a summary of the current challenges to gene therapy and future outlook.

Keywords: β-haemoglobinopathies, CAR T-cell therapy, CRISPR genome editing technology, Gene delivery vectors, Genetic disorders, Gene therapy, Haematological disorders, Haemophilia, Leukaemia, Lymphoma.