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.2024.14.0001
Received: 15 Nov., 2023    Accepted: 20 Dec., 2023    Published: 01 Jan., 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.

Cai X.P., 2024, Applications of gene editing and gene therapy in the treatment of genetic disorders, International Journal of Molecular Medical Science, 14(1): 1-7 (doi: 10.5376/ijmms.2024.14.0001)

 Gene editing and gene therapy are advanced biotechnologies that have gained significant attention in recent years, holding enormous potential for the treatment of genetic disorders. This review provides an overview of the applications of gene editing and gene therapy in the treatment of genetic diseases. It introduces the concept of gene editing and places a specific emphasis on the application of the CRISPR-Cas9 system in genetic disease research and therapy. Furthermore, the review outlines the principles and methods of gene therapy, including gene replacement, gene correction, and gene silencing, discussing their applications in the treatment of genetic disorders. By comparing the advantages and limitations of both gene editing and gene therapy strategies, including technical constraints, safety concerns, and ethical considerations, this review summarizes the current applications of gene editing and gene therapy in the treatment of genetic diseases and provides insights into future trends. The aim of this review is to offer a reference and guidance for research and clinical applications of gene editing and gene therapy in the field of genetic disease treatment.

Gene editing; Gene therapy; Genetic diseases; CRISPR-Cas9; Gene correction