What is Gene Therapy?

Gene Therapy: An Investigational Cure

Gene therapy is being investigated as a cure for Sickle Cell Disease. Gene therapy is the replacement or correction of abnormal genes in order to treat genetic disorders. The gene therapy modifies the patient’s bone marrow cells so that they produce normal red blood cells. The gene therapy patient receives genetically modified cells to replace the damaged cells.

Gene therapy is actively being researched for SCD, but is only available through clinical trials. It holds the promise of cure for those lacking well matched donors.