“A Young Mississippi Woman’s Journey Through A Pioneering Gene-Editing Experiment”

  1. (2) Listen to “A Young Mississippi Woman’s Journey Through A Pioneering Gene-Editing Experiment” (22
    min) https://www.npr.org/sections/health-shots/2019/12/25/784395525/a-young-mississippi-womans-journeythrough-a-pioneering-gene-editing-experiment
    a. Regarding her journey, what would you like to ask Victoria Gray?
    b. Regarding her journey, what would you like to say to Victoria Gray?
  2. For an overview of how CRISPR works, watch this video CRISPR Decoded (2.22 min)
    https://www.technologyreview.com/video/608474/crispr-decoded/
  3. (6) CRISPR-Cas9 Mechanism & Applications. Use this link, then click “Launch Interactive:”
    https://www.biointeractive.org/classroom-resources/crispr-cas-9-mechanism-applications Once the interactive
    figure is open (it may take a while), click on “How it Works.” Scroll down and click on the highlighted terms to
    answer the following questions. Think about your answers and try to learn while going through this.
    a. What kind of enzyme is Cas9? ______________
    b. Describe the purpose of the guide RNA:
    c. What does Cas9 recognize and bind to along the DNA? _____________
    d. What does Cas9 do to the DNA when it binds to the PAM motif?
    e. What has to happen before Cas9 will cut the DNA?
    f. When other enzymes in the cell repair the broken DNA, the repaired DNA often has mutations. Why might a
    scientist want a mutation to remain in a gene?
  4. (2) Watch this video and answer the following questions. CRISPR: Gene editing and beyond (4:31min)
    https://youtu.be/4YKFw2KZA5o
    a. CRISPR-Cas9 is used to cut DNA at specific locations. Cas9 can be modified in different ways. Name two
    other things that the CRISPR-Cas9 system can be used for when Cas9 is modified.
    i. _______________________________________________
    ii. _______________________________________________
  5. (5) Visit Sickle Cell Gene Therapy with CRISPR https://www.synthego.com/crispr-sickle-cell-disease Some
    of the information here is a bit technical, but I think you will be able to find answers to these questions:
    a. Sickle cell disease is caused by a mutation of a single base in the DNA sequence of the gene encoding ßglobin. What oxygen carrying protein is affected by this mutation?

b. In order to have sickle cell anemia (aka sickle cell disease, SSD), does a person have to inherit the mutation
from both parents or just one parent?
c. What is currently the only cure available for sickle cell disease?


d. What are two main ways that CRISPR-Cas9 is being used to treat sickle cell disease?
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i. ____________________________________
ii. ____________________________________
e. Of the two approaches above, which one was used with Victoria Gray?

  1. Any final comments?
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