Genetic Engineering and CAR-T Cell Therapy in Hematological Malignancies: From CD19/BCMA Targeting to CRISPR-Enabled Cellular Engineering

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This research paper explores the rapidly evolving role of genetic engineering and Chimeric Antigen Receptor T-cell (CAR-T) therapy in the treatment of hematological malignancies. It examines major therapeutic targets, particularly CD19 and B-cell maturation antigen (BCMA), and their clinical significance in the management of B-cell leukemias, lymphomas, and multiple myeloma. The paper further highlights the emerging potential of CRISPR-enabled cellular engineering to improve the design, precision, functionality, and therapeutic performance of CAR-T cells. It discusses innovative approaches for enhancing anti-tumor activity, overcoming treatment resistance, developing next-generation allogeneic CAR-T therapies, and improving the safety and persistence of engineered immune cells. The study also addresses important challenges, including cytokine release syndrome, neurotoxicity, antigen escape, tumor heterogeneity, off-target genetic effects, manufacturing complexity, and accessibility. Overall, this work provides an integrated perspective on how CD19/BCMA targeting and CRISPR-based genome engineering are shaping the future of cellular immunotherapy and precision medicine for hematological cancers.