REVIEW article
Front. Med.
Sec. Gene and Cell Therapy
Volume 12 - 2025 | doi: 10.3389/fmed.2025.1690225
The Future of Pediatric Gene Therapy: CRISPR-Cas9, AI, and Personalized Medicine
Provisionally accepted
- 1Jilin Province FAW General Hospital, Changchun, China
- 2Department of Cardiology, FAW General Hospital, Changchun, China
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The future of pediatric gene therapy is being reshaped by the integration of CRISPR-Cas9, artificial intelligence (AI), and personalized medicine, offering unprecedented opportunities to treat rare genetic disorders in children. This article explores how CRISPR-Cas9 enables precise correction of mutations in conditions like sickle cell disease, cystic fibrosis, and Duchenne muscular dystrophy. AI enhances these efforts by optimizing guide RNA design, predicting off-target effects, and tailoring therapies to individual genetic profiles, thereby improving safety and efficacy. We review recent clinical trials, including ex vivo CRISPR-Cas9 applications for hemoglobinopathies and in vivo therapies for retinal disorders, highlighting delivery systems such as adeno-associated viral vectors and lipid nanoparticles. Next-generation tools like base editing and prime editing further refine precision by minimizing double-strand breaks, reducing genotoxicity risks. AI-driven tools, such as DeepCRISPR and CRISPR-GPT, are pivotal in streamlining experimental design and enhancing therapeutic outcomes. The synergy of these technologies enables personalized treatment strategies, addressing the unique genetic makeup of pediatric patients. However, challenges like off-target effects, delivery efficiency, and ethical concerns particularly equitable access and long-term safety remain critical hurdles. This article emphasizes interdisciplinary collaboration to overcome these barriers, advocating for standardized regulations and robust AI integration to accelerate clinical translation. By harnessing CRISPR-Cas9, AI, and personalized medicine, pediatric gene therapy holds transformative potential to provide curative solutions for children with previously untreatable genetic disorders, paving the way for a new era in precision medicine.
Keywords: CRISPR-Cas9, genetic disorders, Rare Diseases, Genome editing, artificial intelligence, personalized medicine
Received: 21 Aug 2025; Accepted: 06 Oct 2025.
Copyright: © 2025 Pan, Ding, Wang, Xu, Fan and Zhang. 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: Wenbin Zhang, zwb80311a@sina.com
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.