Name: ________________________
Gene Therapy Lab Webquest
Date:___________
Instructions: Google search “Utah genetics gene therapy”. Click on the first link. Explore the 3 sections on the left to
answer the following questions.
1) What is Gene Therapy
Most commonly, how does gene therapy work?
Click on Gene Therapy Case Study: Cystic Fibrosis (Section 1). Describe the mutation that causes Cystic Fibrosis.
Click on Gene Therapy Case Study: Cystic Fibrosis (Section 2).
Could the condition be corrected by adding one or a few functional genes?________
Do you know which genes are involved?__________
Do you understand the biology of the disorder?________________________________________________
Will adding a normal copy of the gene fix the problem in the affected tissue?_______
Can you deliver the gene to cells of the affected tissue?_______ Which part?_____________
2) Gene Delivery: Tools of the Trade
First, go to the Tools of the Trade animation (http://learn.genetics.utah.edu/content/tech/genetherapy/gttools/)
This animation will help you understand the various vectors that can be used to insert genes in cells.
For each of the vectors, describe the cells or tissues targeted, what activates the vector, how it integrates, and any possible negative
side effects.
Vector
Target
Activation
Integration
Retrovirus
Adenovirus
Adenoassociated
virus
Herpes
simplex virus
Liposome
Naked DNA
(plasmid)
Click on Gene Therapy Case Study: Cystic Fibrosis (Section 3).
What is the best choice for CFTR gene delivery? __________________________
Side Effects
3) Space Doctor
Now that you have your genetic tool kit, you’re ready to administer gene therapy on three patients. You’re a doctor on a faraway space
station and three patients with genetic disorders come to you for help. Fill out the table for each patient, and record your success.
http://learn.genetics.utah.edu/content/tech/genetherapy/spacedoctor/ Choose 1patient to treat!
Patient name: ___________________________________________
a. What is the patient’s disorder?
b. Summarize the patient’s species characteristics (may be multiple pages).
c. Summarize the disorder characteristics (may be multiple pages).
d. Before you begin, which tissue should you target, and why?
e. Before you begin, which vector will you use to deliver the gene, and why?
f.
What were the results of your gene therapy?
g. If your treatment caused more problems (or if you killed your patient!), try again. What combination of target
tissue and vector worked the best?
Summary: