Scientists with the Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED) at Children’s Hospital of Philadelphia are pioneering work in the field of gene therapy to treat conditions such as sickle cell disease and thalassemia. For people with these conditions, their gene for hemoglobin — the protein that transports oxygen through the body — does not work correctly. Through a procedure called gene addition, gene therapy teaches the hemoglobin to work correctly, so people can live healthy lives.
This animated video explains of how gene therapy works to treat conditions such as sickle cell disease and thalassemia. You'll learn:
- What do our genes do?
- What is hemoglobin?
- How does gene therapy create normal hemoglobin?
- Will gene therapy make a person with sickle cell disease healthy?
Transcript
Gene Therapy for Sickle Cell Disease and Thalassemia
Narrator: Welcome to the CuRED Clinic at the Children's Hospital of Philadelphia. Our scientists are pioneering new work in the field of gene therapy to treat conditions such as sickle cell disease, thalassemia, and more. Genes make us who we are -- our hair color, whether we are short or tall, all the instructions for our bodies to work correctly.
Genes also have the blueprint for making red blood cells and a protein inside them called hemoglobin. Hemoglobin is the protein that transports oxygen. For those of us with sickle cell disease and thalassemia, the gene for hemoglobin is not working correctly. With gene therapy, we can teach the body to make normal hemoglobin.
We collect millions of special patient cells, called blood stem cells, and add a normal copy of the hemoglobin gene to each cell, a procedure scientists call gene addition. We then put these corrected super cells back into the patient's body, where they start to make normal hemoglobin. Your normal hemoglobin will work just as well as that of friends and family members who do not have sickle cell disease or thalassemia. And if your body makes enough hemoglobin, you'll be able to lead a healthy, normal life. For more information about our CuRED Gene Therapy Program, click to visit us online.
Related Centers and Programs: Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED)