Gene and Cellular Medicine Program

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 clinical researchers, infusion center experts and other clinical and research teams work together to ensure a seamless continuum of care for our pediatric patients and their families. 


Our bodies are made of trillions of cells. Cells are like tiny factories that make products the body needs to grow and stay healthy. The instructions for making these products are stored in our genes. Each gene is a very long string of DNA molecules. Sometimes the reason that a person has a health problem is because there is a disease-causing change in one or more of their genes. These harmful genetic changes may cause cell to make a product that just doesn’t work right or even stop cells from making the product at all.

What is gene and cellular therapy?

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

When a gene mutation (a permanent change in the DNA sequence) causes a protein to be missing or faulty, gene and cell therapy may be able to restore the normal function of that protein. There are several different approaches to gene therapy:

  • Introducing a new or modified copy of a gene
  • Turning on or off genes to avoid disease
  • Replacing the disease-causing sequence of a gene with a healthy copy of that sequence

The goal of gene and cellular therapy is to change the course of disease by targeting its genetic cause.

Lurie Children’s offers a variety of FDA-approved therapies for various medical conditions and continues to investigate ways to offer more.

Conditions and 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 was 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 the first FDA approved gene therapy for patients 4-5 with DMD. Elevidys delivers a copy of a gene that has a shortened functional form of dystrophin, the gene that is mutated in DMD patients. The shortened gene will help improve muscle health and slows 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 one of the first FDA approved ocular gene therapies. It is given by injection directly into the eye. Luxturna is approved for RPE65-related diseases such as retinitis pigmentosa or Leber congenital amaurosis. This gene, RPE65, has instructions for making a protein that is needed for normal vision. Luxturna improves vision in individuals with pathogenic variants (gene changes causing the condition) in their RPE65 gene. 

Our Gene & Cellular Therapy Specialists

Vamshi K. Rao, MD

Attending Physician, Neurology

Associate Professor of Pediatrics (Neurology and Epilepsy), Northwestern University Feinberg School of Medicine

Jennifer L. Rossen, MD

Attending Physician, Ophthalmology; Clinical Practice Director; Member, Lurie Children's Surgical Foundation

Assistant Professor of Ophthalmology, Northwestern University Feinberg School of Medicine

Sonali Chaudhury, MD

Section Head, Stem Cell Transplantation and Cellular Therapies

Professor of Pediatrics (Hematology, Oncology and Stem Cell Transplantation), Northwestern University Feinberg School of Medicine

Robert I. Liem, MD

Division Head, Hematology, Oncology, Neuro-Oncology & Stem Cell Transplantation; Director, Comprehensive Sickle Cell Program; Meryl Suzanne Weiss Distinguished Professorship in Hematology, Oncology & Stem Cell Transplantation

Professor of Pediatrics, Northwestern University Feinberg School of Medicine

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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. 

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