Zhaohong Pu , Xiaoping Cai

Xiongcheng Jianmin Med. Ltd., Zhuji, 311800, China
Author    Correspondence author
International Journal of Molecular Medical Science, 2024, Vol. 14, No. 1   doi: 10.5376/ijmms.2024.14.0011
Received: 05 Mar., 2024    Accepted: 05 Apr., 2024    Published: 19 Apr., 2024

This is an open access article published under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Pu Z.H., and Cai X.P., 2024, The progress and challenges of CRISPR-Cas system from laboratory to clinical practice, International Journal of Molecular Medical Science, 14(1): 80-88 (doi: 10.5376/ijmms.2024.14.0011)

The CRISPR-Cas system, as an innovative and efficient gene editing technology, has provided new possibilities for genetic research and disease treatment. This technology draws inspiration from the natural defense mechanisms of microorganisms, enabling precise editing of genes by "cutting" and "pasting" DNA sequences. While it has found extensive applications in laboratory conditions and demonstrated promising results in clinical trials, its transition from the laboratory to clinical applications faces several key issues and challenges, including safety and side effects, ethical and moral considerations, as well as legal and regulatory issues. This review will focus on the progress of CRISPR-Cas system applications from the laboratory to clinical settings, particularly in the realms of gene therapy and disease research. It will extensively analyze and discuss challenges in aspects such as safety, precision, delivery efficiency, aiming to provide meaningful insights and guidance for future research and applications in this field.

CRISPR-Cas system; Gene editing; Gene therapy; Safety; Ethical considerations