Gene Therapy and Precision Medicine in Pediatric Practice

This chapter examines the rapidly advancing field of gene-based and precision therapeutic approaches in pediatric medicine, reviewing the clinical progress of gene therapy for monogenic diseases including spinal muscular atrophy, hemophilia, and lysosomal storage disorders, as well as the application of CRISPR-Cas9 genome editing technologies in experimental and emerging clinical contexts. The chapter also addresses the ethical, regulatory, and health equity dimensions of deploying precision medicine tools in pediatric populations globally.