George 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, $75,000 research grant from the National Blood Foundation to investigate blood disorders.
Murphy’s laboratory studies inherited blood disorders including sickle cell disease, amyloidosis and numerous thrombotic disorders. One goal of Murphy’s research is the development of targeted therapies for these conditions. Research of these disorders has been limited thus far due to a lack of in vitro disease models of disease processes.
“This grant will allow us to utilize a unique, immediately actionable, combinatorial basic and translational research platform to combat inherited blood disease,” said Murphy. “It is our hope that this program will both decipher how these disorders develop via disease modeling and simultaneously address the most serious complication of these diseases, which is the need for frequent blood transfusions.”
The rates of HIV infection associated with blood transfusions, for example, are .003, 0.1 and 0.6 percent in high, middle and low-income countries, respectively, according to data from the World Health Organization. Additionally patients receiving blood product transfusions are at risk for having an immune response to the transfused agent.
Murphy’s laboratory utilizes induced pluripotent stem cells (iPSC) that can develop into any cell type to create models of the inherited blood disorders of interest. They also will work to make use of the iPSC-based system to make an infinite supply of blood cells that could one day be used for transfusions into these patients, which would not be limited by either potential contamination or supply issues.