MEDICAID NOTICE: Lurie Children’s continues to serve all patients enrolled in Medicaid. As a safety-net hospital, we will continue providing high-quality care to every child who needs us.

AVISO SOBRE MEDICAID: Lurie Children’s continúa atendiendo a todos los pacientes inscritos en Medicaid. Como hospital perteneciente a la red de protección social, continuaremos brindando atención de alta calidad a cada niño que nos necesite.

Lurie Children's Gene and Cellular Medicine Program is committed to advancing treatment of conditions that are amenable to gene and cell-based therapy. 

Our program looks to bring hope to patients who live with various blood disorders, neurogenerative conditions, and other genetic disorders. Our integrated team of clinical researchers, infusion center experts, and other healthcare professionals collaborates to provide a seamless continuum of care for our pediatric patients and their families. 

Background

Our bodies consist of trillions of cells, each functioning like miniature factories producing essential products the body needs to grow and stay healthy. The instructions for making these products are stored in our genes. Sometimes, health issues arise due to disease causing changes in one or more of our genes. These genetic changes can cause cells to produce dysfunctional or insufficient amounts of essential products, leading to complicated health issues.  

What is gene and cellular therapy?

Gene and cell therapy is a way to treat or prevent disease. 

When a gene mutation (a permanent change in the DNA) causes certain cells in your body to stop working properly or stop working at all, gene and cellular therapy can potentially restore the normal function to those cells. There are different approaches to gene and cellular therapy: 

  • Introducing a new or modified gene  
  • Regulating the activity of the gene(s) to prevent the disease  
  • Replacing the disease-causing segment of the gene with a health version.  

Lurie Children’s provides a range of FDA-approved therapies for various medical conditions and continually explores new avenues for treatment advancement through clinical studies.  

FDA Approved Treatment Options

Spinal Muscular Atrophy (SMA)

  • Spinraza (Nusinersen): Spinraza was the first FDA approved treatment for spinal muscular atrophy. Spinraza has been approved to treat all patients diagnosed with any type of SMA. In patients with SMA, the SMN1 gene is missing, and the SMN2 gene is the only source of protein production. Spinraza works by targeting the SMN2 gene to promote a more functional protein that is critical for motor neuron cell survival. Spinraza contains an antisense oligonucleotide that increases the ability of the SMN2 gene to produce a full-length SMN protein. The goal is to promote motor neuron health and change the natural progression of spinal muscular atrophy.
  • Zolgensma (Onasemonogene Abeparvovec): Zolgensma is a one-time gene therapy treatment and the second FDA approved treatment for SMA. In May 2019, Zolgensma was approved for all patients with SMA under the age of two. Zolgensma targets the genetic cause of SMA by replacing the missing SMN1 gene. The SMN1 gene is critical to motor neuron cell survival. It is made up of a new SMN gene that is placed inside a viral vector called adeno-associated virus 9 (AAV9). This vector travels through the body to deliver the new gene. Once the new gene has been delivered, the motor neuron cells will now be able to produce SMN protein. 
  • Evrysdi (Risdiplam): Evrysdi, approved by the FDA August 2020 is the third therapy created for those with SMA. It is the only approved SMA treatment that can be taken by mouth or via a feeding tube. Evrysdi works to address the underlying cause of SMA, a low amount of the survival motor neuron protein known as SMN. This therapy is designed to help the body produce more SMN protein throughout the body 

Duchenne Muscular Dystrophy (DMD) 

  • Elevidys (delandistrogene moxeparvovec-rokl): Elevidys is a one-time gene therapy treatment and the first FDA approved therapy for patients 4 years and older with Duchenne Muscular Dystrophy (DMD). Elevidys is designed to strengthen muscles by delivery a shortened version of the dystrophin gene, known as micro-dystrophin. This compensates for the missing or broken dystrophin gene that causes DMD. The shortened gene will help improve muscle health and slow muscle wasting.  

Transfusion Dependent Beta Thalassemia

  • Zynteglo (betibeglogene autotemcel): Zynteglo is the first cell-based gene therapy for patients with beta-thalassemia who require regular red blood cell transfusions. This unique therapy is made specifically for each child, by adding functional copies of the beta-globin gene to their own blood stem cells.  

B-Acute Lymphoblastic Leukemia

  • Kymriah (tisagenlecleucel): Kymriah is a CAR-T cell therapy approved by the FDA to treat children and adolescents with B-acute lymphoblastic leukemia that is resistant to chemotherapy or that has relapsed multiple times. 

Inherited Retinal Diseases

  • Luxturna (Voretigene neparvovec): Luxturna is a one-time gene therapy treatment for inherited retinal disorders caused by mutations in the RPE65 gene. Luxturna delivers a working copy of the RPE65 gene to the retina (the light-sensitive tissue at the back of the eye). This healthy copy helps restore the visual cycle, a series of chemical reactions in the retina in response to light – and the first step in a person’s ability to see. 

Sickle Cell Disease

  • Lyfgenia (lovotibeglogene autotemcel): Lyfgenia is a one-time gene therapy treatment for people aged 12 years and older with sickle cell disease and a history of vaso-occlusive pain crises. This unique therapy uses a vector (delivery vehicle) consisting of a harmless virus to bring working copies of the beta-globin gene. It has been found to decrease or eliminate vaso-occlusive pain crises in many people.
  • Casgevy (exagamglogene autotemcel): Casgevy is a dual indication one-time gene therapy product. Casgevy was approved by the FDA for the treatment of patients 12 years and older with sickle cell disease with recurrent vaso-occlusive crises (VOCs) AND for patients 12 years and older with transfusion dependent beta thalassemia. Casgevy uses a gene editing technology called CRISPR/Cas9 to modify a patient’s own blood-forming stem cells. The treated cells then begin making new, healthy red blood cells.  

Our Gene & Cellular Therapy Specialists

Carlos E. Prada, MD

Division Head, Genetics, Genomics and Metabolism; Medical Director; Cellular and Gene Therapy Program; Valerie and George D. Kennedy Research Professorship in Human Molecular Genetics

Nancy L. Kuntz, MD

Attending Physician, Division of Neurology; Medical Director, Mazza Foundation Neuromuscular Program; Director, Muscular Dystrophy Association Care Center

Jennifer L. Rossen, MD

Attending Physician, Ophthalmology; Clinical Practice Director

Sonali Chaudhury, MD

Section Head, Stem Cell Transplantation and Cellular Therapies

Make an Appointment

  • To make an appointment, visit our Appointments page
  • For any questions, please reach out to our Operations Manager, Adeola Adelabu at genetherapy@luriechildrens.org.  
  • If you are an international patient interested in any of the therapies listed above, please contact our International and Destination Patient Services team at IPS@luriechildrens.org

Resources

Gene Therapy Resources

Condition Resources:

Research/Clinical Trials

Lurie Children’s is dedicated to solving pediatric health challenges through research discoveries and advancements. To learn more about our clinical trials please visit the Stanley Manne Children's Research Institute website. 

Related News

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Gene therapy is a way to correct harmful genetic changes. Its purpose is to give cells the right DNA instructions needed to make products that work as they should.

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Related Specialties

Appointments

If you’d like to request an appointment with one of our specialists, call 1.800.543.7362 (1.800.KIDS DOC®) or visit our Appointments page for more information.