CRISPR in Thalassemia: Global Research Trend Analysis

Area of Science:

• Biotechnology
• Genetics
• Hematology

Background:

• Beta-thalassemia is a common inherited blood disorder affecting beta-globin chains.
• The clustered regularly interspaced short palindromic repeats (CRISPR) genome editing system offers a potential cure.
• Bibliometric analysis is crucial for understanding global research landscapes in emerging fields.

Purpose of the Study:

• To map global research trends in CRISPR-based beta-thalassemia studies.
• To identify key research areas, collaborations, and leading institutions.
• To review CRISPR therapeutic strategies and their translational progress.

Main Methods:

• Bibliometric analysis of Scopus database articles (original and review) from inception to present.
• Search terms included 'beta thalassemia' and 'CRISPR'/'gene editing' variants.
• VOSviewer software used for network visualization of publications, authors, and keywords.

Main Results:

• Significant growth in CRISPR-based thalassemia research, with the US and China as leading contributors.
• Two primary therapeutic strategies identified: direct HBB gene correction and BCL11A repression for fetal hemoglobin reactivation.
• Exagamglogene autotemcel (Casgevy), based on BCL11A repression, received regulatory approval.

Conclusions:

• CRISPR research for thalassemia is transitioning from basic science to clinical application.
• International collaboration is extensive, driving progress in gene editing therapies.
• Future implementation requires advancements in bioprocessing, cost reduction, and adaptable healthcare models.