Gene Therapy and Genetic Engineering

Gene therapy and genetic engineering are rapidly advancing fields in hematology, offering potential cures for inherited and acquired blood disorders. Biologically, these approaches involve correcting or modifying defective genes responsible for conditions such as sickle cell disease, thalassemia, hemophilia, and immunodeficiencies. Techniques include viral vector-mediated gene delivery, CRISPR-Cas9-based gene editing, and RNA-based therapies, which enable targeted modifications of hematopoietic stem cells or immune cells. Diagnostic and laboratory methods are essential for patient selection, monitoring, and evaluating therapy efficacy. These include molecular genetic testing, sequencing, flow cytometry, PCR assays, and functional studies to assess gene expression and protein production. Instruments such as gene-editing platforms, automated cell culture systems, flow cytometers, PCR machines, and high-throughput sequencing instruments support research and clinical applications. Gene therapy allows personalized treatment by restoring normal gene function, improving hemoglobin production, or enhancing immune function. Clinical outcomes have shown remarkable improvements in patients previously dependent on lifelong transfusions or therapies. Safety monitoring is critical, as off-target effects, immune reactions, and vector-related complications must be managed. This session provides delegates with an in-depth understanding of the biological basis, laboratory techniques, instrumentation, and clinical applications of gene therapy and genetic engineering, emphasizing the transformative potential of these approaches in modern hematology.

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