Gene Therapy for Sickle Cell Disease and Thalassemia

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?

 

Related Centers and Programs: Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED)