induced Pluripotent Stem Cells or iPSCs are a form of stem cells that come from skin or blood cells that are changed into cells that have the ability to become any type of tissue in the body. ATTR is a lethal genetic disease in which mutant protein secreted from the liver damages the heart and the brain, highlighting the need for a flexible model system that would allow physicians and scientists to study the disease
According to researchers using this technology, cell lines can be created that are genetically identical to the patient from whom they are derived, allowing for the development of personalized treatments for diseases.
In this study, the researchers used the iPSCs to make liver cells that produce mutant protein as well as cells of the heart and brain, the target tissues of the disease. Upon addition of the mutant protein, the heart and brain cells were damaged, thereby recreating essential aspects of the disease. Furthermore, drugs that are being tested in clinical trials prevented this damage in the cells suggesting that this system can be used to test new drugs for treatment of the disease.
Read the paper here: http://www.cell.com/stem-cell-reports/abstract/S2213-6711(13)00097-0
George J. Murphy, PhD, Assistant Professor of Medicine in the division of Hematology and Oncology at Boston University School of Medicine (BUSM) and co-director of the Center for Regenerative Medicine (CReM) at Boston University and Boston Medical Center has received a two-year research grant from the National Blood Foundation to investigate blood disorders. Read more about the award here: http://www.bumc.bu.edu/2013/07/08/busm-investigator-receives-national-blood-foundation-grant-for-blood-disease-research/