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Integrating Crispr/Cas Technology With Clinical Trials: Principles, Progress and Challenges Publisher



Yang P ; Khoshandam M ; Bhia I ; Raji S ; Soltaninejad H ; Hosseinkhani S ; Sani M ; Hamidieh AA ; Sheykhhasan M
Authors

Source: Asian Journal of Pharmaceutical Sciences Published:2025


Abstract

CRISPR represent a groundbreaking genome-editing technology that has revolutionized genetic modification. This innovative tool offers an unparalleled revolution in the future treatment of genetic disorders, neurological diseases, infectious diseases and cancer. Despite the rapid expansion of CRISPR applications, its clinical use in humans is still relatively limited, with only 69 active clinical trials and 6 completed studies reported so far. This review examined current clinical trials and their processes in addressing various diseases via the CRISPR/Cas system. While earlier literatures have focused mainly on delivery methods and materials for CRISPR/Cas9, our review emphasized innovative targeting conditions and approaches for novel and functional therapeutic designs. In addition, we reviewed recent research to increase the efficiency of CRISPR editing in the management of genetic disorders and cancer, while exploring their future challenges and potential. This review provided a unique perspective on the advancement of CRISPR technology. By addressing these aspects, we aim to contribute to ongoing efforts to improve CRISPR-based therapies and expand their clinical applications, ultimately striving to transform the future of medical treatment. © 2025 Shenyang Pharmaceutical University.
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