Therapeutic Screening Center
Program Leader: Lee L. Rubin, PhD
The HSCI Therapeutic Screening Group was established to take comprehensive advantage of some of the unique aspects of stem cell biology to identify reagents and compounds that either affect cell differentiation or could be therapeutic agents themselves. The lab is equipped with robotic liquid handling systems, a conventional plate reader for standard measurements such as needed for cell-based luciferase assays, and a high throughput confocal microscope-based detector that will allow non-homogeneous differentiation assays (using, for example, GFP or other fluorescent reporters) to be set up. The group has a special interest in orphan nervous system disorders, but also works with other members of the HSCI community and third parties, including those who have a direct interest in translational research.
One way that screening assays use disease-specific cell lines is to look for drugs that could help treat a given disease. The stem cell-based screens pursue this goal by addressing questions on two levels. The first asks mechanistic questions about a specific disease's biology and the second explores how these mechanistic insights can be translated into the search for therapeutic targets.
Screens can also be used to search for drugs or biologics that direct stem cells into becoming a specific type of cell—a process known as targeted differentiation. Knowing how to generate a certain type of cell from stem cells is critical for treating conditions where cells have been injured or died, such as in diabetes.
Questions? - Please contact:Lee Rubin, Ph.D.
Director of Translational Medicine
Bauer Building Cambridge, MA 02138