Demystifying Gene-Based Therapies in Pediatric Health Care

<h2>Abstract</h2> Genomic advancements have enabled the development of therapies that directly target the molecular defects underlying an increasing number of pediatric genetic disorders. These therapies offer the potential for disease-modifying or curative outcomes in conditions that previously lacked effective treatments. Many health care professionals, including nurse practitioners, may be unfamiliar with the rapidly developing field of gene therapy in pediatrics. This report explores the clinical application of gene-based therapies using 2 clinical examples, spinal muscular atrophy and sickle cell disease, both of which use different techniques to tackle the genetic defect at its root.