Global Research Trends and Hotspots in Gene Editing and Stem Cell Therapies for Neurodegenerative Diseases:...

Neurodegenerative diseases are a major and growing global health burden. Their pathogenesis is complex, and effective therapies remain limited. Gene editing and stem cell-based strategies are reshaping the therapeutic landscape. However, the field has not been systematically examined through bibliometric analysis.

We aimed to define the intellectual landscape of global research on gene editing and stem cell therapy for neurodegenerative diseases from 2005 to 2024, highlight evolving hotspots, track the field's evolution, and identify major bottlenecks limiting clinical translation.

We retrieved 1821 publications from the Web of Science Core Collection (2005-2024). We performed a multidimensional bibliometric analysis using CiteSpace and VOSviewer. We assessed publication output, country and institutional contributions, key authors and journals, co-cited references, and keyword networks. These analyses were used to track the field's evolution and pinpoint emerging themes.

In total, 9978 researchers from 90 countries and 2515 institutions contributed to this literature. Annual publications increased from 28 in 2005 to 179 in 2024, with stepwise growth over time. The United States ranked first in output (n=780) and in citation impact (total local citation score=2784; total global citation score=40,009). China and India ranked second and fifth in output, respectively, but their average citation impact was lower than that of the leading countries. The University of California, San Francisco, and Johns Hopkins University remained consistently influential. Boulis NM, Bankiewicz KS, and Feldman EL were among the most prominent contributors. Molecular Therapy was the leading journal in this area. Keyword analyses pointed to a growing intersection between genetics and immunology. Major topics included nanotechnology-based delivery, adeno-associated virus vectors, small interfering RNA, intrathecal microsphere injection, autophagy, blood-brain barrier (BBB) targeting, clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), and induced pluripotent stem cells. Burst detection highlighted "open label" as a recent hotspot. This likely reflects rising translational activity and early clinical testing.

The field is moving from technology development toward clinical translation. Anglo-American countries currently drive both productivity and influence. China and India contribute heavily to volume but need a stronger impact. CRISPR/induced pluripotent stem cell platforms and BBB-focused delivery remain central frontiers. The rise of "open-label" studies suggests accelerating clinical momentum. Future progress will require safer and more efficient delivery, clearer standards, and larger global consortia to harmonize protocols and speed translation.