Undifferentiated stem cell library to be created for sickle cell disease
Researchers are creating an induced pluripotent stem cell library containing blood samples from individuals with sickle cell disease.
Scientists from the Center for Regenerative Medicine at Boston University School of Medicine (MA, USA) and Boston Medical Center (MA, USA) are establishing a stem cell library from ethnically diverse individuals who have sickle cell disease. Described in Stem Cell Reports, the resource is being created to emphasize the importance of a library for future research and therapy development.
Sickle cell disease is an inherited red blood cell disorder that causes abnormal hemoglobin in blood and is carried by approximately 5% of the world’s population. Researchers are planning to use the undifferentiated induced pluripotent stem cells (iPSCs) in the library to create disease models in the hope of obtaining new insights about the disease and developing new tests and treatments.
George Murphy, cofounder of the Center for Regenerative Medicine (Boston University School of Medicine) explained, "sickle cell disease affects millions of people worldwide and is an emerging global health burden…iPSCs have the potential to revolutionize the way we study human development, model life-threatening diseases and, eventually, treat patients."
One of the project leaders, Gustavo Mostoslavsky (Boston University School of Medicine), commented: "In addition to the library, we've designed and are using gene editing tools to correct the sickle hemoglobin mutation using the stem cell lines…When coupled with corrected sickle cell disease specific iPSCs, these tools could one day provide a functional cure for the disorder."
In the future, the stem cell library is hoped to become an invaluable resource where the stem cell lines are available to researchers worldwide. Martin Steinbery (Boston University School of Medicine) concluded: "With their philosophy and guiding principles, the results that they share could help speed up the process of finding novel treatments and potentially cures for diseases affecting people across the world."