Bibliometric Insights into CRISPR-Cas9 Research Targeting Human Disease Pathology

CRISPR-Cas9 has rapidly emerged as a transformative genome editing platform. To analyze and map scientific research on CRISPR-Cas9 in the context of human disease pathology. A bibliometric study was conducted on original research articles indexed in Scopus between 2010 and 2024, focused on CRISPR-Cas9 applications in human disease pathology. A total of 4,313 publications were identified with an average annual growth rate of 54.5% and an H-index of 146. The top publishing journals were Scientific Reports, International Journal of Molecular Sciences , and Nature Communications. The United States led with 1,727 articles, followed by China ( n = 1,355), with 50.9% international collaboration for the United States versus 34.4% for China. The average number of authors per article was 10.8. The top-cited article reported on CRISPR-Cas9 therapy for beta-thalassemia and sickle cell disease, followed by major studies on cystic fibrosis, amyloidosis, muscular dystrophy, and colorectal cancer. The following research themes were identified: (1) cancer pathways and metastasis (2) viral genome targeting; (3) mutation-driven genetic disorders; and (4) hematological disorders. VOSviewer maps indicated that off-target effects, delivery methods, and ethical concerns are major challenges for CRISPR-Cas9 use in human diseases. The analysis reflects exponential scientific growth and translational momentum. Future research will require refined delivery strategies, safety optimization, and ethical integration to fully harness CRISPR-Cas9’s clinical potential. Clinical Trial Registration : Not applicable.