Question

A team of researchers from Vertex Pharmaceuticals and Crispr Therapeutics have initiated this limited clinical trial to treat two patients with blood disorders using a revolutionary gene-editing technology. Victoria is being treated for sickle cell anemia and another patient is being treated for β-thalassemia.What is the current treatment for sickle cell anemia? Is this treatment effective at preventing patients’ from experiencing symptoms of the disease?

Answer 1

Sickle cell anemia is a disorder of the blood caused by an inherited abnormal hemoglobin. The abnormal hemoglobin causes distorted Red blood cells. It is an autosomal recessive disease.  

Bone marrow transplant, also known as blood cell transplant, offers the only potential cure for sickle cell anemia. Finding a donor is difficult, and the procedure has serious risks associated with it, including death. It is usually reserved for people younger than age 16, because above it risk increases.

It involves replacing bone marrow affected by sickle cell anemia with healthy bone marrow from a donor. The procedure usually uses a matched donor, such as sibling who does not have sickle cell anemia, if donor unavailable, stem cells from umbilical cord can be used.

If a donor is found, the person with disease receives radiation or chemotherapy to destroy or reduce his/her bone marrow stem cells. Healthy cells from donor are injected intravenously into the bloodstream of the person with disease, where they migrate to the bone marrow and begin generating new blood cells.

The procedure requires a lengthy hospital stay. After the transplant, patient receives drug to help prevent rejection of the donated stem cells.