CRISPR/Cas9 in red blood cell-related diseases: breakthroughs in gene editing and therapy

Le résumé fourni par la source

Red blood cell (RBC) disorders, including sickle cell anemia and β-thalassemia, are emerging as a significant global public health concern. Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9)-mediated genome editing technology, commonly referred to as CRISPR/Cas9, is a precise and efficient genome-editing tool that has demonstrated considerable potential in advancing research on erythroid diseases. This review elucidates the molecular mechanisms underlying CRISPR/Cas9 technology and systematically illustrates its applications in generating animal models for RBC disorders, investigating erythrocyte differentiation pathways, and developing novel therapeutic strategies. Furthermore, the review critically evaluated the current challenges and future directions for CRISPR/Cas9-based approaches, providing insights to guide both fundamental research and clinical translation in the context of these hematologic conditions.

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