At the Forefront - UChicago Medicine

Gene Therapy and Transplant for Sickle Cell Disease

At UChicago Medicine Comer Children’s Hospital, sickle cell disease patients have access to groundbreaking treatment options, including gene therapy and stem cell transplant. Our team of experts are at the forefront of sickle cell disease research and collaborate with each patient to develop individualized treatment plans unique to their needs.

Advanced Sickle Cell Disease Treatment Options

Gene therapy

New sickle cell gene therapies were approved by the Food and Drug Administration in 2023 and represent a major step forward for sickle cell treatment.

What is sickle cell gene therapy?

Both types of gene therapy involve removing a patient’s own cells and modifying them before infusing them back into the patient.

One type of sickle cell gene therapy involves gene addition, which adds a new copy of a gene to your stem cells so they produce a functional form of hemoglobin.

The other type of sickle cell gene therapy involves gene editing, which changes your existing DNA to “switch on” a gene that will make your stem cells revert to producing fetal hemoglobin instead of non-functional adult hemoglobin affected by the sickle cell mutations.

Comer Children’s was one of the first hospitals in the US to be activated as an authorized treatment center for Vertex Casgevy CRISPR-Cas9 gene therapy and will also offer Bluebird Lyfgenia gene therapy.

Comer Children's among the first in the country to offer newly approved sickle cell gene therapies

Stem cell transplant

This is when healthy blood stem cells are transplanted into the patient’s body, replacing the bone marrow with blood cells that make normal hemoglobin instead of sickle hemoglobin.

Learn more about stem cell transplant

Frequently Asked Questions About Sickle Cell Gene Therapy and Transplant

Meet Our Sickle Cell Gene Therapy and Stem Cell Transplant Physicians

Our gene therapy and stem cell transplant experts, led by Dr. James LaBelle, are at the forefront of the latest sickle cell treatments and research.

We partner with Comer Children's sickle cell disease specialists to offer advanced treatment options for individuals with the disorder.

During your gene therapy or stem cell transplant journey, you will receive care from a wide range of pediatric specialists and support services who work together to provide personalized care for each patient.

Meet our care team

James LaBelle, MD, PhDJames LaBelle, MD, PhD

Pediatric Hematology & Oncology (Pediatric Cancer)

John M. Cunningham, MDJohn M. Cunningham, MD

Pediatric Hematology & Oncology (Pediatric Cancer)

Saara Kaviany, DOSaara Kaviany, DO

Pediatric Hematology & Oncology (Pediatric Cancer)

Adam DuVall, MD, MPHAdam DuVall, MD, MPH

Pediatric Hematology & Oncology (Pediatric Cancer)

Philip Connell, MDPhilip Connell, MD

Radiation Oncology

Request an Appointment

The information you provide will enable us to assist you as efficiently as possible. A representative will contact you within one to two business days to help you schedule an appointment.

To speak to someone directly, please call 773-702-6808. If you have symptoms of an urgent nature, please call your doctor or go to the emergency room immediately.

For Referring Physicians

To refer a pediatric patient, please call UCM Physician Connect at 1-800-824-2282. 

By submitting this form you acknowledge the risk of sending this information by email and agree not to hold the University of Chicago or University of Chicago Medical Center liable for any damages you may incur as a result of the transfer or use of this information. The use or transmittal of this form does not create a physician-contact relationship. More information regarding the confidentiality of this request can be found in our Privacy Policy.

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By submitting this form you acknowledge the risk of sending this information by email and agree not to hold the University of Chicago or University of Chicago Medical Center liable for any damages you may incur as a result of the transfer or use of this information. The use or transmittal of this form does not create a physician-contact relationship. More information regarding the confidentiality of this request can be found in our Privacy Policy.