MINI REVIEW article
Front. Genome Ed.
Sec. Genome Editing in Human Health and Disease
Therapeutic Applications of CRISPR-Cas9 Gene Editing
Provisionally accepted
- 1Green Level High, Cary, United States
- 2National Center for Genome Resources, Santa Fe, United States
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CRISPR-Cas9 is a gene editing tool used extensively in biological research that is now making its way into clinical therapies. With the first CRISPR therapy obtaining approval by the United States' Food and Drug Administration (FDA) in late 2023, we look at clinical trials of emerging therapies involving CRISPR-Cas9, currently the most prevalent CRISPR-based tool in these trials. A CRISPR-based therapy is currently approved for treatment of both sickle-cell anemia and transfusion-dependent β-thalassemia but clinical trials for CRISPR-based therapeutics include a much broader range of targets. CRISPR-Cas9 is being explored to treat cancer, infectious disease, and more. This review highlights CRISPR-Cas9 clinical trials registered at clinicaltrials.gov as of 12/31/2024.
Keywords: CRISPR-Cas9, gene editing, Therapeutics, clinical trials, Cancer
Received: 13 Oct 2025; Accepted: 29 Nov 2025.
Copyright: © 2025 Bharti and Mudge. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Joann Mudge
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.