Researchers at the Children’s Hospital of Philadelphia make progress on sickle cell disease treatment.
Sickle cell disease still persists 100 years after discovery.
A new way to repair Sickle Cell?
Researchers have recently developed a safer way to repair sickled cells in a patient’s body. Traditionally, bone marrow or stem cell transplants are used but pose the significant risk of rejection by the patient’s immune system. A new method involves the use of the patient’s own cells. The skin cells are induced to become a specific kind of stem cell called induced pluripotent stem cells, or iPSC’s. These cells are especially important because they can develop into any bodily tissue, including blood. Ideally, scientists can take these stem cells and “fix” the DNA to essentially remove the mutation causing Sickle Cell Disease. While this research is very promising, many issues are still being worked out. Read the full article below.
Safe Way to Repair Sickle Cell Disease Genes, Study Suggests
A study has been recently published that scientists hope will help with treatment of Sickle Cell Disease. A device has been introduced that would be used to “guide the timing of clinical interventions, to monitor the progression of the disease, and to measure the efficacy of drugs, transfusion, and novel small molecules in an ex vivo setting.” The primary purpose of the device is to quantitatively measure the effects of vaso-occlusion, or cell “jamming,” that is the major effect of Sickle Cell Disease. This is a novel discovery because it will allow for doctors to be able to better detect and monitor a patient’s condition, and coordinate appropriate treatment. Below is the link to the actual study:
A Biophysical Indicator of Vaso-occlusive Risk in Sickle Cell Disease