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New Ways to Breathe

Cystic Fibrosis and Gene Therapy

Co Authors:

Charlie W. Zhao
School of Medicine
Yale University

Kevin Xo

Western University

Tom L. Haffie
Department of Biology
Western University


This case study follows a young cystic fibrosis (CF) patient named Lucas. Through Lucas's story and interactions between his parents and pediatrician, students learn about the scientific background and basis of CF. By reviewing email correspondence between Lucas's parents and various doctors, students gain an overview of CF research. CF has become a model disease in certain undergraduate biology classrooms due to its relatively clear mechanism and genetic basis. This case asks students to come up with their own ideas to improve on an existing line of research - gene therapy - in treating CF. During the process, students will gain a better appreciation of the innovative nature of science and develop research skills such as finding, understanding and analyzing primary literature. The activity was originally designed for first- and second-year students as part of an extracurricular case competition, but may be used for any undergraduate biology level. The case assumes basic (high school level) knowledge of genetics, biochemistry, cell biology and physiology.

  • Describe the genetic and cellular basis of CF.
  • Describe the basic mechanism of gene therapy and its challenges.
  • Access, and derive meaning from, the scientific literature in order to identify further avenues of research to explore.
  • Summarize a selected area of CF research.
  • Demonstrate the creative and collaborative nature of scientific inquiry by working with peers to formulate original solutions within the given constraints.
  • Convey scientific information and ideas effectively in writing.
Keywords: cystic fibrosis; CF; gene therapy; CFTR; autosomal recessive disorder; mucus;
Topical Area: N/A
Educational Level: High school, Undergraduate lower division, Undergraduate upper division
Formats: PDF
Type/Method: N/A, Directed, Discussion
Language: English
Subject Headings: Biochemistry   Biotechnology   Cell Biology   Medicine (General)   Molecular Biology   Pharmacy / Pharmacology   Physiology   Public Health  
Date Posted: 4/10/2017
Date Modified:
Copyright: Copyright held by the National Center for Case Study Teaching in Science, University at Buffalo, State University of New York. Please see our usage guidelines, which outline our policy concerning permissible reproduction of this work.

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The following video(s) are recommended for use in association with this case study.

  A Cure for the Colorblindness Blues “Science
Using a virus-based gene therapy and a group of highly trained monkeys, Maureen and Jay Neitz may have created a cure for colorblindness. Created by: Science Friday. Produced by: Luke Groskin. Running Time: 5:58 min. Date: September 1, 2017.

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