Awakening Fetal Hemoglobin Genes with CRISPR-Cas9

The chapter explores using CRISPR-Cas9 technology to reawaken dormant fetal hemoglobin genes as a potential therapy for blood disorders like sickle cell disease and beta thalassemia. It discusses the advancements in gene editing techniques such as base editing and prime editing for precise and stable DNA corrections, emphasizing the importance of minimizing unintended consequences. The ethical considerations and strategic partnerships in the development of gene editing technology for therapeutic purposes are also highlighted.