The Pompe Predicament

Lesson 1 | Lesson 2 | Lesson 3 | Lesson 4 | Lesson 5 | Lesson 6 | Lesson 7 | Lesson 8

BCS Graphic 2


Pompe disease affords the rare opportunity for students to consider multiple biological concepts and assemble them into a story.  Instead of DNA taught separate from protein structure and function, these areas as well as enzymes, genetics and human disease are all taught together through the story of Pompe disease.  If you are currently using sickle cell disease or cystic fibrosis in your curriculum, you may want to consider adding Pompe to your toolbox! You can preview the Web-based Inquiry Science Environment (WISE) module of this curriculum here.

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In the introduction to The Pompe Predicament you will find tips on using the curriculum, summaries of each lesson, a lesson sequencing guide, pertinent vocabulary, a standards alignment chart (Next Generation Sunshine State Standards), and general background information for the unit.

Introduction to the Pompe Predicament

Lesson 1: Looking Through a Father’s Eyes  

A first person story is presented to the students to hook their interest in the disease. Using a jigsaw approach, students will learn about the fundamentals of Pompe disease and share information during a whole class discussion.   We often know the symptoms and pathology of diseases and perform a differential diagnosis to determine which disease fits the symptoms, then proceeding with treatment accordingly. In this lesson, students will understand the basis of the disease, know what cellular structures are affected, deduce the symptoms and propose treatment.  
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Lesson 2: The Road to Treatment

Working in groups, students will read Pompe fact cards and use text clues to sequence the events in the discovery and treatment of Pompe disease. This lesson illustrates scientific discovery as a collaborative effort of many individuals building on prior knowledge and developing unique ideas to explore.

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 Lesson 3: Pompe Journal Club

Journal Club

Using a reading guide, students work in pairs to read a journal article and share their interpretations during a whole class activity. After completing the introductory reading activity, students utilize a reading guide to work independently to read and identify key information before sharing their understanding with others in a small group. If time allows, students translate their paper into a poster to share with classmates during a gallery walk and subsequent poster presentation.

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Lesson 4: From DNA Structure to Function

struc to fxn

With a commercially available kit from Science Take-Out, students step through the process of transcribing and translating DNA sequence. Using the student worksheet provided here, student then consider how the acid alphaglucosidase gene is affected by mutations and how the change in structure affects the function of the enzyme.

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Lesson 5: Putting it All Together

Road to Treatment

Students perform a colorimetric assay on patient samples to determine % activity of GAA, and then perform a confirmatory test by comparing the patient DNA sequence to the reference and identifying any mutations. They will transcribe their sequence into mRNA and translate into amino acids. They will use the sequence along with chenille stems and beads to create the amino acid chain and fold their protein. Comparing the mutant protein to the normal protein, students will infer the functionality of the mutant protein and predict the form of Pompe disease.

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Lesson 6: Grand Rounds

grand rounds

Building on the previous activity, students are given the case report for their patient. They will present a summary of the case along with the lab results they found in a grand rounds fashion. This activity allows students the opportunity to read actual patient cases and work together to summarize the findings. When they present their summary to the class, each member of the group is responsible for presenting part of the case.

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Lesson 7: Exploring Gene Therapy Clinical Trials through Webquest and Role Play

explore gene therapy

In this lesson, students will learn about the benefits, dangers, and ethical dilemmas associated with gene therapy clinical trials through a webquest. They will then participate in a role play, depicting various stakeholders at an RAC meeting to decide the fate of a gene therapy trial. This activity serves as a wrap-up to the entire unit.

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Lesson 8: Extraordinary Measures video guide


After students have a deep understanding of the science of Pompe disease, students view the movie Extraordinary Measures, based on the book The Cure which tells the story of the Crowley family and their battle against Pompe disease. Students complete a movie guide during the film and consider the accuracy of the Hollywood depiction drawing from their scientific knowledge of Pompe disease.

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