A genetic disorder is a disease caused by one or more missing or changed (mutated) genes. Each cell in the human body has thousands of genes, which carry instructions for how cells should work. A missing or mutated gene changes how cells work, which can lead to various genetic disorders, depending on which genes are affected.

The Pediatric Gene Therapy Program at Children’s Health℠ provides advanced gene therapy treatment for children with certain genetic disorders. Gene therapies are medical techniques that can fix or replace missing or mutated genes so that they work properly. These treatments can change the course of certain genetic disorders, keeping them from getting worse or stopping them completely.

The advanced gene therapies we offer are based on decades of research at Children’s Health, UT Southwestern and other research centers. Our gene therapy teams include doctors and providers who both participate in this research and provide patient care. Our comprehensive approach means that your child receives care from a team with years of experience in providing exceptional care for children with rare, complex genetic disorders.

These disorders cause symptoms that gradually get worse, leading to severe disability and even early death, depending on the disorder. Now, gene therapies for some of these disorders provide children with life-changing treatment options, instead of treatment that only manages symptoms. At Children’s Health, we’re dedicated to helping your child live as healthy as possible – for as long as possible.