Current trends of clinical trials involving CRISPR/Cas systems

Songyang Zhang; Yidi Wang; Dezhi Mao; Yue Wang; Hong Zhang; Yihan Pan; Yuezeng Wang; Shuzhi Teng; Ping Huang

doi:10.3389/fmed.2023.1292452

Current trends of clinical trials involving CRISPR/Cas systems

Front Med (Lausanne). 2023 Nov 10:10:1292452. doi: 10.3389/fmed.2023.1292452. eCollection 2023.

Authors

Songyang Zhang^#¹, Yidi Wang^#², Dezhi Mao^#², Yue Wang³, Hong Zhang², Yihan Pan³, Yuezeng Wang¹, Shuzhi Teng¹, Ping Huang¹

Affiliations

¹ The Key Laboratory of Pathobiology, Ministry of Education, Norman Bethune College of Medicine, Jilin University, Changchun, China.
² The Third Affiliated Hospital of Jilin University, Changchun, China.
³ The Second Affiliated Hospital of Jilin University, Changchun, China.

^# Contributed equally.

Abstract

The CRISPR/Cas9 system is a powerful genome editing tool that has made enormous impacts on next-generation molecular diagnostics and therapeutics, especially for genetic disorders that traditional therapies cannot cure. Currently, CRISPR-based gene editing is widely applied in basic, preclinical, and clinical studies. In this review, we attempt to identify trends in clinical studies involving CRISPR techniques to gain insights into the improvement and contribution of CRISPR/Cas technologies compared to traditional modified modalities. The review of clinical trials is focused on the applications of the CRISPR/Cas systems in the treatment of cancer, hematological, endocrine, and immune system diseases, as well as in diagnostics. The scientific basis underlined is analyzed. In addition, the challenges of CRISPR application in disease therapies and recent advances that expand and improve CRISPR applications in precision medicine are discussed.

Keywords: COVID-19; CRISPR; HIV; cancer; clinical trials; gene therapy; genome editing; hemoglobinopathies.

Publication types

Review

Grants and funding

The author(s) declare financial support was received for the research, authorship, and/or publication of this article. This work was supported by the National Natural Science Foundation of China (grant no. 31771624), the Science Technology Commission of Jilin Province (grant no. 20180101145JC) and the Health Technology Innovation Program of Jilin Province (grant no. 2019J011).