Specialties & Services

Gene Therapy

The Gene Therapy and Advanced Therapeutics Program brings together the expertise of WashU Medicine physician-scientists with the specialized hospitals, pharmacy services, and multidisciplinary care teams of BJC HealthCare. Together, we provide advanced treatment options and coordinated support for people with genetic, neuromuscular, immunologic, hematologic, and oncologic disorders, helping you make informed decisions about your care. 

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Gene therapy specialties and services

We blend compassion with leading-edge expertise to deliver advanced treatments and support your health for years to come. 

Cancer
Gene therapy can be used in some forms of cancer to improve the immune system response or change the genes inside cancer cells to stop its growth.

Ear, nose, and throat

 Gene therapy may be used to treat disorders like recurrent respiratory papillomatosis by helping the body's immune system target cells infected by human papillomatosis virus (HPV). For people with hereditary hearing loss, gene therapy may be used to modify the genes in the inner ear to improve hearing.

Hematology

 Gene therapy can be used as a treatment option for several blood disorders—including sickle cell disease and beta-thalassemia—typically by changing the way your body makes blood cells.

Neuromuscular

 People with genetic muscle and nerve disorders, such as spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD), can receive gene therapy to deliver healthy genes into the body to improve muscle and nerve function.

Pediatric

 In addition to providing stem cell transplants, specialists at St. Louis Children’s Hospital also use gene therapy to treat several neuromuscular and genetic conditions. If you want more information about gene therapy for your child, please speak with your pediatrician about a referral to St. Louis Children’s Hospital. If you are a referring provider, you can call Children’s Direct at 800-678-4357.

Stem cell transplant

Gene therapy can be used to modify a person’s own stem cells before a bone marrow transplant to correct genetic issues, slow, and—in some cases—cure certain cancers and blood disorders.

Why choose us for gene therapy?

Building on decades of research and innovation, WashU Medicine and BJC HealthCare bring advanced gene therapies close to home. The Gene Therapy and Advanced Therapeutics Program was established in 2019 and has expanded to include neuromuscular, stem cell transplant, hematology, oncology, and ear, nose, and throat conditions. At BJC HealthCare and WashU Medicine you can expect: 

  • Multidisciplinary care: Many disorders affect multiple parts of the body. You will work with a range of providers across specialties so you can access expert care for your health concerns. 
  • Access to the newest developments: Through the expertise of WashU Medicine physicians at St. Louis Children's Hospital, patients have access to one of the nation's few academic medical center programs offering gene therapy for sickle cell disease. The team was the first in the region to provide this innovative treatment. 
  • Leading-edge expertise: Together with WashU Medicine, the Gene Therapy Program was one of the first in the U.S. to treat patients with Zolgensma®, a landmark gene therapy for spinal muscular atrophy, when it was FDA approved in 2019. This means you can expect to work with a team that has proven experience in treating people with complex gene therapies. 

  • Comprehensive care: Care goes beyond your gene therapy treatment. Our providers offer personalized care before, during, and after your treatment, so you feel mentally and physically prepared for all possible outcomes. 

     

Clinical trials

WashU Medicine researchers and scientists are doing groundbreaking research to find new ways to use gene therapy to treat illnesses that were once thought to be untreatable. Through clinical trials, you have access to new medications and innovative treatments. Read about these innovations and ongoing research and reach out to your provider to see if you’re a candidate for clinical trials.

Discover more about clinical trials

Locations
outside view of Barnes-Jewish hospital

Barnes-Jewish Hospital

One Barnes-Jewish Hospital Plaza
St. Louis, MO 63110
314-747-3000 More about Barnes-Jewish Hospital
Outside view of St. louis Children's Hospital

St. Louis Children’s Hospital

One Children's Place
St. Louis, MO 63110
314-454-6000 More about St. Louis Children’s Hospital