Sickle cell disease is a disabling and painful genetic blood disorder that primarily affects African-Americans. Fortunately, researchers at John Hopkins have found that there are many advantages of adult stem cells being used as a treatment for the disease.
The stem cell treatment corrects the alteration that causes sickle cell disease. The corrected stem cells were persuaded into immature red blood cells in a test tube and then turned into a regular version of the gene.
According to the stem cell research team, the work conducted in the laboratory is years away from being used to treat clinical patients. However, the advantages of adult stem cells therapies are that they provide tools for the development of gene therapies for sickle cell disease as well as a number of other blood disorders.
Researchers believe that they are yet another step in the right direction toward finding a realistic cure or a possible long-term option for treating patients with sickle cell disease. The disease is the result of a single letter change in the human gene responsible for adult hemoglobin, which is the primary protein in red blood cells that are necessary for the transporting of oxygen throughout the body.
When a person inherits two copies of the genetic alteration, which means they get one from each of their parents, the red blood cells take on a sickle shape as opposed to the usual round shape of everyone else’s red blood cells. Because the cells are misshaped, they clog the blood vessels, leading to fatigue, pain, organ damage, infection and even premature death.
Even though painkillers and other drugs are available to treat the symptoms of sickle cell disease, the only known cure is a bone marrow transplant. However, the majority of patients with sickle cell disease are African-American and only a few African-Americans are listed in the bone marrow registry. Therefore, it is difficult to find suitable donors.
Researchers at the John Hopkins Hospital isolated a single patient’s bone marrow cells and reprogrammed theses adult stem cells to act like embryonic stem cells. They then used a normal copy of the hemoglobin gene to replace the one that is defective through the advantages of adult stem cells engineering technologies.
The researchers used three hundred different samples of iPS cells to sequence the DNA to identify the ones that had the right copies of the hemoglobin gene and they only found four. However, three of the iPS cells did not pass assemblage in the following tests.
According to the professor of medicine and associate director of basic research for the Division of Hematology and a member of the John Hopkins Institute for Cell Engineering, Linzhao Cheng, Ph.D., “The beauty of iPS cells is that we can grow a lot of them and then coax them into becoming cells of any kind, including red blood cells.”