Cystic Fibrosis: The Cause, The Treatment

BCH 2105H

Most people with Cystic Fibrosis in Canada and the US have access to an effective therapy called Trikafta. This triple combination of small molecules targets the molecular defects induced by the major mutation, F508del.  This outcome reflects the success of a basic research path leading from gene discovery in 1989 to the determination of protein structure and function relationships. Consequently, an understanding of the consequences of disease-causing mutations enabled the discovery of small molecule modulators of these protein defects. However, 10-30 percent of patients harbouring this mutation do not benefit from Trikafta clinically.  Hence, the basic research in this field is striving to define the cellular and molecular barriers to therapeutic efficacy with the goal of discovering mechanisms to improve in-vitro and clinical outcomes for all affected individuals.

The instructors in the course will discuss progress in defining cellular and molecular determinants of CFTR and F508del-CFTR protein expression and function and hence, potential therapeutic targets.

Non-Biochemistry Students:  This courses will be available to all non-Biochemistry students to request beginning December 1, 2024.  Please contact carrie.harber@utoronto.ca

Students will not be permitted to drop a 0.25 module course if you have completed more than 1 class without approval of the course coordinator(s).

Course Next Offered

Winter 2025

Course Time and Location

9-11 am Thursdays, starting January 9th.

Location: Gilgan building (20-9701)

Enrollment Limit

Yes — 12

Method of Student Evaluation

40%: Twenty minute presentation of peer-reviewed publication from selected list.
40%: Three page critique of the same publication.
10%: Role as modulator
10%: Class participation

Coordinator

Christine E. Bear

Christine E. Bear

The Hospital for Sick Children
Peter Gilgan Centre for Research and Learning
686 Bay St.
416-813-5981
bear@sickkids.ca

Instructors

Last Updated 6 January 2025