The Translational Science of Stem Cells: Present and Future

SCRB 195 Subject & Catalog Number

Description

Information about the biology of stem cells and their uses in understanding and treating diseases -- particularly those that cannot be studied adequately in non-human model systems -- has increased enormously in the last decade. In this seminar/lecture course, students will learn about transplanting functional human cells (such as pancreatic beta cells or dopaminergic neurons) derived from pluripotent cells to treat disease. They will also discover how to use these cells to model diseases, such as neurodegenerative and cardiovascular diseases, with the goal of identifying more effective, possibly patient-specific, therapeutics. Students will hear about treatments, including small molecules (conventional medicines), whose therapeutic actions can be attributed to the regulation of tissue-specific stem cells that reside in key adult tissues including the bone marrow and brain, but, interestingly, not including the heart or pancreas. Finally, they will be exposed to relatively new work that demonstrates the possibility of creating new cells from old by using genetic methods to swap cell identities. A typical type of question that we will debate is: When should Parkinson’s disease patients be treated with a drug to slow the death of neurons, with transplanted neurons made from pluripotent cells or with a viral vector that produces new neurons from existing glial cells in the brain? 

This course will highlight the theoretical, as well as the practical, aspects of drug development. How are therapies progressed from conception to patient (bench to bedside)? How can academic investigators commercialize research? Importantly, while this is a science course, not a health economics course, we intend to discuss ways of reducing drug costs. At the same time, we will introduce the new trend of treating rare (even N=1) genetic diseases and how this is or isn’t accommodated within our existing healthcare framework. 

We believe that students with different backgrounds (biology, chemistry, engineering, business) and at different levels (undergraduate, graduate) can benefit from taking the course and will help enrich the discussions by providing different perspectives on topics that we’ll cover. However, basic knowledge of cell and molecular biology will be needed to understand the course fully.

Course Notes

Permission of the instructor is required to enroll for students who have not taken the courses below. Ability to work in a less structured environment will be essential, as will the ability to work with other students. A pass-fail option is not available in this course.

Recommended Prep

Life Sciences 1a or Life and Physical Sciences A, Life Sciences 1b, and preferably SCRB 10 or NEURO 80.