Cystic Fibrosis: The Cause, The Treatment - BCH2105H

This course will examine molecular and cellular aspects of the causes and the treatment for cystic fibrosis (CF) disease. The first three two-hour sessions of the course will consist of lectures by Dr. Charles Deber and Dr. Christine Bear, while the latter three two-hour sessions will consist of oral presentations by the students.

Dr. Deber’s lectures will focus on the cystic fibrosis transmembrane conductance regulator (CFTR) – the membrane protein that is defective in CF patients – and how mutations in CFTR affect its structure and function; and aspects of the lung infections that result from CFTR defects, including formation of bacterial biofilms, and treatment with currently-available antibiotics.

Dr. Bear will discuss the consequences of CF-disease-causing mutations on biosynthesis, function and stability in various cell models and in patient-derived tissues (including tissues differentiated from pluripotent stem cells); the importance of such patient-oriented research in revealing the important role of genetic modifiers in determining CF disease severity; and the recent development of mutation-targeted therapies in treating CF and the first role of stem cell biology and genomic medicine in creating the next generation of more effective interventions.

Method of Student Evaluation

Each student will select a journal article from a list of recent papers relating to the above topics, and present the paper in oral and written form.
- 50% on the student’s oral presentation of the selected paper, including critical review/assessment of its key findings
- 50% on a subsequent written version of his/her critique (maximum 5 pages double-spaced) that will be due after the last class

Course Coordinator:
Christine Bear

Enrollment Limit:
12
A minimum of 6-8 students is required for this course to run.