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Social determinants of health and access to allogeneic hematopoietic cell transplantation, megakaryocyte growth factor receptor-based pretransplant conditioning for ex vivo autologous gene therapy, and novel protein biomarkers for risk stratification in a

Blood Podcast

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Advancements in Ex Vivo Gene Therapy and Targeted Cell Depletion

This chapter explores innovative ex vivo autologous gene therapy strategies for treating inherited blood disorders like beta-thalassemia and sickle cell disease. It highlights the critical role of a conditioning regimen and introduces a new immunotoxin aimed at enhancing stem cell engraftment and discusses promising preclinical results.

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