Gene Therapy in Pediatrics

Gene therapy in pediatrics involves treating children's genetic disorders by correcting or replacing faulty genes responsible for their condition. This is typically done by introducing healthy copies of genes into the child's cells using harmless viruses or other methods. The goal is to restore normal function or halt disease progression, offering potential cures or improved quality of life. It is a promising area of medicine that addresses childhood conditions like inherited blood disorders or certain metabolic diseases, with ongoing research aimed at increasing safety and effectiveness for young patients.