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Activity 2.2.1: Gene Therapy Introduction In Lesson 2.1, you explored the science behind genetic testing and screening. You saw how DNA sequencing technology and bioinformatics have opened the door to identifying specific genes in our genome. Faulty genes are often at the heart of devastating illnesses and disorders. But what if scientists could penetrate our cells and replace a gene that is malfunctioning? With a working copy of the gene, the proper protein could be produced and the disease could be eliminated. It may sound like science fiction, but scientists have already opened the door to this molecular treatment known as gene therapy. Gene therapy is the practice of inserting functional genes into a person’s genome to replace faulty genes. Doctors and medical researchers are working to restore function to cells that are affected by genetic disorders. To accomplish this goal, scientists have to get the right gene to the right cells in the right tissue. This is not an easy task. Many vectors, or delivery systems, for gene therapy are actually viruses. The idea of using viruses to deliver treatment may sound frightening, but these tiny pathogens can be used as an effective way to target our cells and insert new genes. In this activity, you will investigate how gene therapy works and learn about the vectors currently being investigated as possible transport systems for new genes. You will read current research on gene therapy trials and gather evidence for and against this type of disease treatment. Ultimately, you will develop a policy for regulating and controlling future gene therapy research. Equipment Computer with Internet access and Inspiration® software Activity 2.2.1 Student Response Sheet Laboratory journal Procedure Part I: Mechanics of Gene Therapy 1. With a partner, brainstorm diseases that you feel could be cured using gene therapy. Refer back to the genetic testing scenarios from Lesson 2.1 and to genetic disorders you discussed in PBS for ideas. Identify the faulty gene in at least one of the diseases on your list. 2. Visit the Genetic Science Learning Center (GSLC) Gene Therapy site at http://learn.genetics.utah.edu/content/tech/genetherapy/ and read the three sections listed below. Take notes in your laboratory journal. © 2010 Project Lead The Way, Inc. MI Activity 2.2.1 Gene Therapy – Page 1 o What is Gene Therapy? o Choosing Targets for Gene Therapy o Gene Delivery: The Key to Gene Therapy 3. Use Inspiration software to create a concept map summarizing what you have learned about gene therapy. Include the disease and the faulty gene you identified in Step 1 as an example. 4. Answer Conclusion question 1. 5. Return to the GSLC site and open the Tools of the Trade interactive. 6. Obtain a Student Response Sheet from your teacher. 7. Note that for gene therapy to be successful, a DNA delivery system, or vector, is necessary. Click the toolbox to explore possible vectors for gene therapy. Record information about these vectors on the Student Response Sheet. 8. Answer Conclusion question 2. 9. Use the information you have compiled on your Student Response Sheet to complete the GSLC Space Doctor interactive found at http://learn.genetics.utah.edu/content/tech/genetherapy/spacedoctor/. Choose and treat one patient. Note that the interactive may take a few minutes to load. As you complete the interactive and treat your patient, think about the following questions: o Will the gene of interest fit into the vector? o Will the vector target the right type of tissue? o What type of cells does the therapy need to target (dividing/nondividing)? 10. Document the case from the interactive in your laboratory journal and describe the evidence you used to choose the proper vector. Make sure to list the patient by name and provide a description of the fictional condition, including information about the gene affected, the cells in question and the overall prognosis of the patient. If you choose a vector that does not work, explain why it did not work in your laboratory journal and describe how you altered your treatment plan. 11. Answer Conclusion questions 3 and 4. Part II: Debating Gene Therapy 12. Form a group of four. 13. Two members of the group will choose and read four of the following articles describing evidence against the use of gene therapy. o The New York Times – Patient Dies During a Trial of Therapy Using Genes http://query.nytimes.com/gst/fullpage.html?sec=health&res=9E 06EED8173EF93AA1575AC0A96F958260 o Fox News – Boy Living in Bubble Develops Leukemia From Experimental Therapy http://www.foxnews.com/story/0,2933,317225,00.html © 2010 Project Lead The Way, Inc. MI Activity 2.2.1 Gene Therapy – Page 2 o Newsweek – Juice for Today’s Athlete: Is ‘Gene Doping” the Next Olympic Threat? http://www.newsweek.com/id/151988/output/print o The New York Times – 2nd Cancer is Attributed to Gene Used in FDA Test http://query.nytimes.com/gst/fullpage.html?res=9801E7DB1F31 F934A25752C0A9659C8B63 o ABC News – Patient Death Latest Setback for Gene Therapy http://abcnews.go.com/Health/Story?id=3421869&page=1 o The New York Times – Trials are Halted on a Gene Therapy http://query.nytimes.com/gst/fullpage.html?res=9C01E7D9163B F937A35753C1A9649C8B63 14. The two remaining members of the group will choose and read four of the following articles arguing in favor of gene therapy: o Biology News Net – Gene Therapy Shows Promise as Weapon Against HIV http://www.biologynews.net/archives/2009/02/23/gene_therapy_ shows_promise_as_weapon_against_hiv.html o New Scientist - Gene Therapy Cures “Bubble Boy” http://www.newscientist.com/article/dn2124-gene-therapycures-bubble-boy.html o The New York Times – More Than Human http://www.nytimes.com/2005/07/03/books/chapters/0703-1stnaam.html o New Scientist – Gene Therapy is First Deafness ‘Cure’ http://www.newscientist.com/article/dn7003 o Stanford University Medical Center – Gene Therapy for Muscular Dystrophy Fixes Frail Muscle Cells in Animal Model http://www.eurekalert.org/pub_releases/2005-12/sumcgtf122205.php o USA Today – Gene Therapy Experiments Improve Vision in Nearly Blind http://www.usatoday.com/tech/science/2008-04-283714044875_x.htm 15. Print out the articles you have selected if instructed to do so by your teacher. 16. Read the articles and record evidence in your laboratory journal that supports the viewpoint you were asked to research. © 2010 Project Lead The Way, Inc. MI Activity 2.2.1 Gene Therapy – Page 3 17. Present your evidence to your group members while the other pair takes notes in their laboratory journals. Switch roles and take notes as the other pair presents their evidence. 18. Have a discussion with the other pair debating the pros and cons of gene therapy. Argue for the opposing viewpoint of the articles that you were assigned. Use only the notes that you took as your partners presented their evidence. 19. When both sides have been argued, come to a consensus as to whether gene therapy research should be continued. As a group, write a two to three paragraph statement regarding what you believe to be effective policy for gene therapy research. Should research continue? If so, with what restrictions? If you decide research should not be continued, explain why and provide possible alternatives. 20. Explain your group’s decision and policy to the class. 21. Use the Internet to find an article about gene therapy being used for a disease, disorder or condition NOT discussed in this activity. Bullet key points of the article in your laboratory journal and share your findings with the class. 22. Answer the remaining Conclusion questions. Conclusion 1. How do doctors and researchers decide whether a disease is a good candidate for gene therapy? 2. What are the advantages and disadvantages of using viral vectors for gene therapy? 3. What factors must be considered when choosing a vector for a gene therapy trial? © 2010 Project Lead The Way, Inc. MI Activity 2.2.1 Gene Therapy – Page 4 4. A young boy suffers from Neurofibromatosis Type 1 (NF1), a genetic nervous system disorder that causes tumors to grow around nerve cells. The gene for NF1, approximately 8,400 base pairs long, is located on chromosome 11. What is the best candidate for a vector for gene therapy in this case? Provide evidence from your research to support your choice. 5. Many athletes and administrators fear the widespread use of gene doping – the use of gene therapy to modify genes that improve athletic performance. Describe at least two genetic modifications that would improve the performance of an athlete. Make sure to mention the body system(s) affected as well as the specific goal of the therapy. 6. Do you believe gene doping should be considered cheating? Why or why not? © 2010 Project Lead The Way, Inc. MI Activity 2.2.1 Gene Therapy – Page 5 7. Why do you think testing for gene doping would be extremely difficult? 8. Explain why some physicians and clinicians feel that gene therapy is a distant future consideration. © 2010 Project Lead The Way, Inc. MI Activity 2.2.1 Gene Therapy – Page 6