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/

A study led by Boston University School of Medicine has identified a novel approach to create an unlimited number of human red blood cells and platelets in vitro. In collaboration with Boston University School of Public Health (BUSPH) and Boston Medical Center (BMC), the researchers differentiated induced pluripotent stem (iPS) cells into these cell types, which are typically obtained through blood donations. This finding could potentially reduce the need for blood donations to treat patients requiring blood transfusions and could help researchers examine novel therapeutic targets to treat a variety of diseases, including sickle cell disease.  Published online in the journal Blood, the study was led by George J. Murphy, PhD, assistant professor of medicine at BUSM and co-director of the Center for Regenerative Medicine (CReM) at Boston University and BMC and performed in collaboration with David Sherr, PhD, a professor in environmental health at BUSM and BUSPH.

Murphy Lab members Amy Leung, Sarah Rozelle and Shirley Nah visit Manchester Essex Regional High School to teach students about stem cell biology and regenerative medicine, inspiring the youth of today to pursue careers in the sciences.

Sarah Rozelle, a graduate student in the Murphy lab, is profiled on the Boston University School of Medicine webpage…click on the link to read more…

Shirley Nah, a Master’s student in the Murphy Lab, was presented a prestigious research award by David Coleman for her work involving the induced pluripotent stem cell modeling of neurodegenerative disease.

Sarah Rozelle, a 3rd year graduate student in the Murphy Lab was selected to give an oral presentation of her work at the 18th annual Globin Switching meeting held at the Asilomar Conference Center, Monterey, CA.