Graduate Student Janet Hsu Awarded WORLD Scholarship
Janet Hsu, a graduate student in the Fischell Department of Bioengineering at the University of Maryland (BIOE), was recently awarded a scholarship from the World Organization for Rare Lysosomal Disorders (WORLD) scholarship to attend the 8th annual meeting in San Diego, CA. Her award-winning abstract, titled “Enhanced Kidney and Heart Delivery of α-Galactosidase by Modulating Enzyme Load and Carrier Bulk-Concentration of ICAM-1-Targeted Nanocarriers” was one of only ten that were selected for this honor. Ms. Hsu is a predoctoral student working in the laboratory of Dr. Silvia Muro, Assistant Professor in the University of Maryland’s Institute for Biotechnology and Bioscience Research and BIOE.
Ms. Hsu’s research focuses on Fabry disease, a lysosomal storage disorder that results in a clinical condition affecting multiple tissues. Lysosomes are organ-like intracellular structures that contain enzymes necessary for metabolizing the cell’s food sources, such as lipids, carbohydrates, and other proteins. Fabry disease results when gene mutations affect an enzyme, α-galactosidase A (α-Gal), involved in the metabolism of certain glycolipids in the body. Clinical enzyme replacement therapy (ERT) by injection of recombinant α-Gal improves the disease outcome somewhat, but has shown limited effects on the vascular pathology that is a hallmark of the most life-threatening complications of the disease. In the past, Dr. Muro’s lab has reported on a strategy to improve vascular delivery of α-Gal. To achieve this, they loaded α-Gal in polymer nanocarriers that were targeted to intercellular adhesion molecule 1 (ICAM-1), a protein expressed on the endothelium throughout the vasculature. This novel delivery system enhanced enzyme delivery in mice and biochemical effects in cell models. The work described in Ms. Hsu’s abstract for the WORLD scholarship explored the impact of several nanocarrier design parameters in the biodistribution and final enzyme delivery in mice, to optimize therapeutic doses for future pre-clinical testing.
The work in Dr. Muro’s lab focuses on mechanisms of endocytic vesicular transport. This includes a translational application for the controlled delivery of nano-scale therapeutics to precise targets at the sub-cellular level, such as the work presented by Ms. Hsu. Other projects in the Muro lab include a project, recently funded by the NIH Chemical Genomics Center (NCGC), to combine a platform that Dr. Muro’s lab has developed for crossing the blood-brain barrier with a number of new therapeutic agents identified by her NCGC collaborators. The research is sponsored by grants from the National Institutes of Health and the American Heart Association.