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Cell and Gene Therapy: Research Progress and Future Directions


HAO Sha1,2,3, CHENG Tao1,2,3*

(1State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China; 2Tianjin Institutes of Health Science, Tianjin 301600, China)
Abstract:

CGT (cell and gene therapy) has evolved over nearly several decades from basic research to clinical applications. Significant advances have been achieved in the areas such as cell therapy, gene editing, and delivery vector technology, opening new therapeutic avenues for blood diseases, cancers, autoimmune diseases, genetic diseases, and degenerative disorders. However, challenges remain, including technical complexity, safety concerns, and high manufacturing costs. This review systematically categorizes CGT-based pharmaceuticals and summarizes their clinical trial progress. This review conducts a comparison of the status and differences in CGT drug development between China and the global landscape, analyzes current bottlenecks, and discusses future directions. The aim is to provide a comprehensive update for researchers in the field, promote further development of CGT, and propose innovative treatments to improve outcomes for many refractory diseases.



CSTR: 32200.14.cjcb.2026.01.0001