Sometimes it is All in the Genes
Gene Therapy Assignment
by Anne Galbraith and David Howard
Department of Biology, University of Wisconsin—La Crosse

Do some research on one of the genetic disorders we explored earlier (bottom of Part 3).  Be prepared to present your response to the following question:  Having weighed the pros and cons of gene therapy for your genetic disorder, decide whether or not you would enroll Joshua in clinical research trials for gene therapy for your disorder?  Justify your decision!  Your presentation should be based on your team's research into the learning issues we identified in class.  At the very minimum, your presentation should also address the following questions:

  1. Current therapies (non-gene therapy) available to treat genetic disorders only treat the symptoms of the disorder.  However, if gene therapy were to work, it could be considered a cure rather than a mere treatment of the symptoms.  How could this be the case for many genetic disorders?
  2. What is the state of the current research on gene therapy for treating genetic disorders in general? 
  3. Is gene therapy a possible cure for the genetic disorder your team chose? Why or why not? Explain your answer. What is the state of research on gene therapy for your team's disorder? 
  4. Describe one procedure that might be used for treating Joshua through gene therapy (for your disorder).  
  5. If gene therapy is not an option for Joshua (for your disorder), is there something that someday could be done to prevent his children from inheriting the disorder?
Below are some starting resources on general information about gene therapy!  Journal articles should be accessible throught the College of Charleston Library. 
Use your textbook too!