Progress and Perspective of CRISPR-Cas9 Technology in Translational Medicine.

ABSTRACT: Translational medicine aims to improve human health by exploring potential treatment methods developed during basic scientific research and applying them to the treatment of patients in clinical settings. The advanced perceptions of gene functions have remarkably revolutionized clinical treatment strategies for target agents. However, the progress in gene editing therapy has been hindered due to the severe off-target effects and limited editing sites. Fortunately, the development in the clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR-Cas9) system has renewed hope for gene therapy field. The CRISPR-Cas9 system can fulfill various simple or complex purposes, including gene knockout, knock-in, activation, interference, base editing, and sequence detectio

SUBMITTER: Zheng R 

PROVIDER: S-EPMC10477906 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature