New Ways to Breathe
Cystic Fibrosis and Gene Therapy
School of Medicine
Department of Biology
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.
Keywordscystic fibrosis; CF; gene therapy; CFTR; autosomal recessive disorder; mucus;
Educational LevelHigh school, Undergraduate lower division, Undergraduate upper division
Type / MethodsN/A, Directed, Discussion
Subject HeadingsBiochemistry | Biotechnology | Cell Biology | Medicine (General) | Molecular Biology | Pharmacy / Pharmacology | Physiology | Public Health |
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The following video(s) are recommended for use in association with this case study.
- A Cure for the Colorblindness Blues
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.