About Sickle Cell Disease (SCD):
- It is a group of inherited blood cell disorders that affect hemoglobin, the molecule in Red Blood Cells (RBCs) that delivers oxygen to cells throughout the body.
- SCD can cause episodes of severe pain and lead to life-threatening complications.
- The most common and severe type of SCD is sickle cell anemia.
- How Does it Affect Blood Flow?
- Normally, RBCs are disc-shaped and flexible enough to move easily through the blood vessels.
- People with SCD have atypical hemoglobin molecules called hemoglobin S, which can distort RBCs into a sickle, or crescent, shape.
- When RBCs sickle, they do not bend or move easily and can block blood flow to the rest of the body.
- The sickle-shaped cells can also stick to vessel walls, causing a blockage that slows or stops the flow of blood.
- What causes it?
- The cause of SCD is a defective gene, called a sickle cell gene.
- A person will be born with SCD only if two genes are inherited—one from the mother and one from the father.
- If born with one sickle cell gene, it's called sickle cell trait. People with sickle cell trait are generally healthy, but they can pass the defective gene on to their children.
- Treatments:
- A bone marrow transplant (stem cell transplant) can cure SCD.
- However, there are treatments that can help relieve symptoms, lessen complications, and prolong life.
- Gene therapy is also being explored as another potential cure.
- The UK recently became the first country to approve gene therapy treatment for SCD.
