Gene Therapy and Genome Editing in Non-Malignant Hematological Disorders

This chapter reviews the remarkable recent progress in gene therapy and genome editing approaches for hemoglobinopathies, primary immunodeficiencies, and other inherited disorders of the blood and bone marrow. It covers the clinical development and regulatory approval of gene therapy products for sickle cell disease and beta-thalassemia, including lentiviral vector-based and CRISPR-Cas9-based strategies targeting BCL11A and the HBG gene loci. The chapter also addresses challenges related to genotoxicity, manufacturing scalability, long-term durability of response, and global equity of access to these transformative but costly therapies.