Researchers Make Progress in Finding Cure for Sickle Cell Disease

Health Wellness

Image result for sickle cell anemia

Right off the bat, I’m going to shatter a common held myth about sickle cell disease – black people are NOT the only ones with this disease. It also affects people from (and the descendants of people from) the Arabian Peninsula, Italy, Greece, Turkey, India, Spain (and many Spanish speaking countries), the Caribbean, Central and South America. It seems that sickle cell disease is common in most areas that are also affected by malaria.

If this includes you and your family, then you may want to pay attention to the following.

Yes, people of black origin have the highest incidence of sickle cell disease but it is not exclusive to just them. Here in the United States, about 1 of every 365 black Americans have sickle cell disease, but did you know that about 1 of every 1,000 to 1,400 Hispanic Americans also have sickle cell disease? The incidents among those of Greek, Italian, Turkish, Indian and Arabian descent have much lower rates of sickle cell, but be aware that these ethnic groups are not immune to the genetic disorder.

Sickle cell refers to a group of disorders that affect the hemoglobin in a person’s blood. This is caused by a genetic mutation that results in the production of hemoglobin S, which causes normally round red blood cells to form in a sickle or crescent shape. As you know, we get half of our genetics from our father and half from our mother and for sickle cell to fully manifest in someone, they must receive the trait from both parents, consequently, about 1 of every 13 black babies born carry the trait from at least one of their parents.

The condition generally shows up in children, but continues on into adulthood. When the red blood cells begin to form a sickle shape, it results in less oxygen carried in the blood, the cells break down easier resulting in anemia, fatigue, and shortness of breath. The sickle-shaped red blood cells are also stickier and tend to stick to each other forming clots in smaller arteries and vessels. This causes the blood flow to extremities to be reduced, a condition that is often quite painful.

Progress in treating sickle cell disease has been made and since 1999, the mortality rate among children 4 or younger fell by 42%, but it’s just treatment, not a cure.

Gang Bao, a bioengineer at Rice University announced that his team has made a major breakthrough in finding a cure for sickle cell disease. He explains:

“Sickle cell disease is caused by a single mutation in the beta-globin gene (in the stem cell’s DNA). The idea is to correct that particular mutation, and then stem cells that have the correction would differentiate into normal blood cells, including red blood cells. Those will then be healthy blood cells.”

His team used gene editing to fix the mutation in 20% to 40% of stem and progenitor cells that were taken from the peripheral blood of people with sickle cell disease. The repaired cells are then injected into the bone marrow of the patient. Most of a person’s supply of red blood cells are created in the bone marrow.

The process is still in the early phase of experimenting and testing, but does offer the first major hope of finding a cure for the most common inherited blood disorder in America. If his work continues to show good results, it could be the best offer of hope for so many who suffer from such a debilitating disorder.

Sickle Cell Anemia

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