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BIO 25Q: Cystic Fibrosis: From Medical Conundrum to Precision Medicine Success Story

An Immune Cell showing how microgravity mimics aginh. NASA International Space Station, 04/21/14, Nasa's Marshall Space Flight Center via Flickr.

General Education Requirements

Way SMA


Course Description

The class will explore cystic fibrosis (CF), the most prevalent fatal genetic disease in the US, as a scientific and medical whodunit. Through reading and discussion of medical and scientific literature, we will tackle questions that include: how was life expectancy with CF increased from weeks to decades without understanding the disease mechanism? Why is the disease so prevalent? Is there an advantage to being a carrier? Is CF a single disease or a continuum of physiological variation—or—what is a disease? How did research into CF lead to discovery of the underlying cause of most other genetic diseases as well?

Through critical reading of the scientific and medical literature, class discussion, field trips and meetings with genetic counselors, caregivers, patients, physicians and researchers, we will work to build a deep understanding of cystic fibrosis, from the biochemical basis to the current controversies over pathogenic mechanisms, treatment strategies and the ethics and economics of genetic testing and astronomical drug costs.


Meet the Instructor: Ron Kopito

Ron Kopito

Ron Kopito is a professor and a cell biologist in the Department of Biology.  He’s a graduate of Bowdoin College and received his Ph.D. from MIT.  His lab uses human genetic disease as a platform to understand basic questions about molecular “quality control”—how our cells can tell a good protein from a bad one and how they destroy the latter. He’s been working on cystic fibrosis since he was in high school.

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