ReviewPediatric research2026
The future of pediatric gene therapy: CRISPR-Cas9, AI, and personalized medicine.
Review in Pediatric research, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The future of pediatric gene therapy is being fundamentally reshaped by the convergence of CRISPR-Cas9 genome editing, artificial intelligence (AI), and personalized medicine, providing unprecedented opportunities for the treatment of rare and previously intractable genetic disorders in children. CRISPR-Cas9 enables precise and potentially curative correction of pathogenic mutations underlying conditions, such as sickle cell disease, cystic fibrosis, and Duchenne muscular dystrophy, with particularly notable progress in hematological disorders. AI-driven tools, like DeepCRISPR and CRISPR-GPT are emerging as valuable assets for streamlining experimental design, prioritizing candidate edits, and accelerating preclinical-to-clinical translation. In parallel, advances in delivery systems, including adeno-associated viral (AAV) vectors and non-viral platforms such as lipid nanoparticles (LNPs), are expanding the feasibility of both ex vivo and in vivo pediatric applications. The synergistic integration of CRISPR-Cas9, AI, and personalized medicine enables more precise, patient-specific therapeutic strategies that better account for the developmental, physiological, and genetic heterogeneity of pediatric populations. Addressing these challenges will require interdisciplinary collaboration across genome engineering, AI science, clinical pediatrics, bioethics, and regulatory policy. The establishment of standardized regulatory frameworks, accompanied by robust and transparent AI integration, will be essential to ensure safe, equitable, and scalable translation. IMPACT: The future of pediatric gene therapy is being reshaped by the integration of CRISPR-Cas9, AI, and personalized medicine. We review recent clinical trials, including ex vivo CRISPR-Cas9 applications for hemoglobinopathies. This article emphasizes interdisciplinary collaboration to overcome these barriers. Pediatric gene therapy holds transformative potential to provide curative solutions for children.
Identifiers
42469418What Socratic holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.