CRISPR, AI and Precision Medicine Converge for Children

Rather than presenting new experimental data, this review synthesises recent developments in paediatric gene therapy. It describes how CRISPR–Cas9 is being applied to correct disease-causing mutations responsible for disorders including sickle cell disease, cystic fibrosis and Duchenne muscular dystrophy, with the strongest clinical progress to date in haematological diseases through ex vivo genome editing.

The authors also discuss the growing role of AI-based tools, such as DeepCRISPR and CRISPR-GPT, in guide RNA design, candidate prioritisation and optimisation of preclinical research, with the aim of improving the translation of genome-editing therapies into clinical practice. Advances in delivery technologies, including adeno-associated viral vectors and lipid nanoparticles, are reviewed as important enablers of both ex vivo and in vivo applications.

The review concludes that integrating CRISPR, AI and personalised medicine could support more patient-specific therapies for children, but emphasises that progress will depend on coordinated work across genome engineering, clinical medicine, bioethics and regulatory science, alongside robust standards for the safe and transparent use of AI.

The study was led by Yinping Pan and Wenbin Zhang at Jilin Province FAW General Hospital, Changchun, China. It was published in Pediatric Research on 17 July 2026.