Hemoglobin

Global Research Trends in Using CRISPR to Treat Thalassemia

Updated

Abstract

CRISPR-based thalassemia research shows a significant transition towards clinical applications, with major contributions from the United States and China.

  • The bibliometric analysis indicates a growing body of research focused on CRISPR strategies for treating β-thalassemia.
  • Two key therapeutic approaches are identified: correcting the gene in hematopoietic stem cells and reactivating fetal hemoglobin through repression.
  • The approval of exagamglogene autotemcel (Casgevy) highlights a significant advancement in the application of CRISPR for this disorder.
  • Emerging techniques such as base editing and prime editing may improve treatment precision and long-term effectiveness.
  • International collaboration is a vital component driving the expansion of CRISPR research in thalassemia.

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