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Salehihikouei G, Rostami Maskopaii S, Gholami A. Gene Therapy for Rare Pediatric Genetic Disorders: A Narrative Review of Strategies and Challenges. J. Pediatr. Rev 2026; 14 (3) :251-262
URL: http://jpr.mazums.ac.ir/article-1-847-en.html

Background: Rare pediatric genetic disorders are a substantial global health burden, with limited treatment options. Gene therapy has emerged as a promising technique to target the causes of these disorders.
Objectives: This review study aims to summarize the current strategies and challenges in gene therapy for rare pediatric genetic disorders, focusing on clinical applications and future prospects. 
Methods: This is a narrative review. A comprehensive literature search was conducted in PubMed, Scopus, Web of Science, and Google Scholar for relevant studies published from 2010 to 2026, using keywords such as “gene therapy,” “rare pediatric diseases,” “viral vectors,” and “CRISPR.” Relevant articles focusing on clinical applications, efficacy, and safety challenges were selected and analyzed to summarize current strategies and future perspectives.
Results: Gene therapy advancements have been made using viral vectors (e.g. adeno-associated virus (AAV) and lentiviral vectors), and genome editing tools such as the CRISPR-Cas system. These therapies have shown success in treating numerous diseases such as neuromuscular diseases and metabolic disorders. Despite promising results, challenges related to immune responses, off-target effects, and the need for long-term follow-up still remain.
Conclusions: Gene therapy offers great promise for treating rare pediatric genetic diseases, but clinical challenges such as safety and treatment durability exist. New innovations in vector design, immune management, and CRISPR-based genome-editing technologies can enhance the effectiveness of these therapies.