Emerging Cell and Gene Therapies in Pediatric Surgery
Keywords:
pediatrics, cell therapy, gene therapy, regenerative medicine, pediatric surgery, CAR‑T, tissue engineering, minimally invasive, rare diseaseAbstract
Cell and gene therapies are rapidly transforming the management of congenital and acquired pediatric surgical diseases by enabling mechanism-based, often potentially curative interventions beyond conventional surgery and pharmacotherapy. This narrative review summarizes recent advances in pediatric cell and gene therapies relevant to surgical practice, with emphasis on monogenic disorders, pediatric cancers, and organ and tissue regeneration. Key platforms include in vivo and ex vivo gene therapy, chimeric antigen receptor (CAR) T cells, stem cell–based regenerative approaches, and their integration with minimally invasive and transplant surgery. We compare therapeutic modalities regarding mechanism, indications, efficacy, and safety, and highlight current challenges in vector delivery, immune responses, cost, manufacturing, and ethical considerations, particularly in neonates and infants. A descriptive synthesis of recent clinical and translational studies is presented, including an illustrative distribution of emerging therapy categories. Finally, we outline priorities for future research, regulatory innovation, and multidisciplinary care models needed to safely translate these powerful therapies into routine pediatric surgical practice
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