Uploaded September 2024 | Updated September 2026, 8 hours ago
It is important to understand the components of gene therapy, how these components differ and other treatment options for sickle cell disease, such as bone marrow transplant. A variety of approaches target the blood stem cells to reduce your symptoms of sickle cell disease. Gene therapies use your own stem cells, while bone marrow transplants use the stem cells of a matched donor.
John Tisdale, M.D., Senior Investigator and Director in the Cellular and Molecular Therapeutics Laboratory at the National Heart, Lung, and Blood Institute, explains the types of gene therapy (which include gene addition and gene editing) and the two types of gene editing: gene silencing and gene correction.
About the Sickle cell disease gene therapy education project: genome.gov/research-at-nhgri/Projects/Democratizing-Education
It is important to understand the components of gene therapy, how these components differ and other treatment options for sickle cell disease, such as bone marrow transplant. A variety of approaches target the blood stem cells to reduce your symptoms of sickle cell disease. Gene therapies use your own stem cells, while bone marrow transplants use the stem cells of a matched donor.
John Tisdale, M.D., Senior Investigator and Director in the Cellular and Molecular Therapeutics Laboratory at the National Heart, Lung, and Blood Institute, explains the types of gene therapy (which include gene addition and gene editing) and the two types of gene editing: gene silencing and gene correction.
About the Sickle cell disease gene therapy education project: genome.gov/research-at-nhgri/Projects/Democratizing-Education
