Pediatric Gene Therapy Program

Groundbreaking clinical research

Our gene therapy doctors are also scientists who engage in research every day, working to find treatments for severe genetic disorders. In close partnership with UT Southwestern, we work with research groups and patient organizations across the country on research projects for a growing number of genetic conditions.

We’re contributing to breakthroughs in pediatric gene therapy as research expands and evolves into new gene-correction techniques. Our researchers are studying dozens of genetic disorders, and many therapies are entering preclinical and clinical research phases. Some of our ongoing research includes gene therapies for:

• CLN7, a form of Batten disease

• Giant axonal neuropathy

• Rett syndrome

• Spastic paraplegia 50

Advanced care across disciplines

In recent years, several gene therapies have successfully completed the clinical trial process. Those therapies have received approval from the U.S. Food and Drug Administration (FDA) as safe, effective treatments. At Children’s Health, eligible patients have access to these gene therapies through our Neurology Department and the Center for Cancer and Blood Disorders.

Learn more about our FDA-approved gene therapies for:

• Blood disorders, including sickle cell disease and beta thalassemia: Casgevy, Lyfgenia, Zynteglo

• Neurological disorders, including cerebral adrenoleukodystrophy, Duchenne muscular dystrophy, metachromatic leukodystrophy and spinal muscular atrophy: Elevidys,  Zolgensma and Skysona.

Extensive experience in caring for children with rare pediatric conditions

Our teams have extensive experience in providing complete care for children with extremely rare, complex genetic conditions. That’s why Children’s Health has been designated as a qualified treatment center for all available FDA-approved gene therapies for children.

Our doctors and providers have years of experience in treating children with these therapies as they went through research studies. Your child’s care team includes doctors and other providers from multiple specialties, including neurology, hematology (blood diseases), oncology (cancer), neuropsychology, nursing and physical therapy. We provide comprehensive care to support your child before they receive gene therapy. And we carefully monitor them after gene therapy for complications of the genetic disorders and treatment side effects.

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.