Genetics Published January 2018 www.BioInteractive.org Page 1 of 2 Short Film Student Handout Genes as Medicine NAME_______________________ DATE_________________ This handout supplements the short film Genes as Medicine. 1. True/False. Soon after a gene associated with childhood blindness was discovered in the 1990s, Drs. Bennett and Maguire were able to offer a gene therapy treatment for children. _______ 2. Explain the reasoning or evidence you used to answer question 1. 3. In the film you met Molly, who has a form of childhood blindness called Leber amaurosis (also called Leber congenital amaurosis, or LCA). Her blindness is caused by _______ a. early exposure to intensely bright light. b. an eye injury she received at a young age. c. mutations in a gene that is necessary for maintaining sight. d. a viral disease in her nervous system that moved into her eyes. 4. For many genetic diseases, children inherit the disease-causing mutations from their parents. What was the likely inheritance pattern in the case of Molly’s form of childhood blindness? _______ a. Only females can have Leber amaurosis, so Molly must have inherited the genetic mutation only from her mother. b. Molly became blind from new mutations that occurred in her eye as a baby. c. Molly ended up with childhood blindness because both of her parents were blind when they were children. d. Molly inherited a mutation in the same gene from each of her parents. 5. Which statements are reasons that eyes are a good target for clinical trials related to genetic medicine? _______ I. Eyes are easy to access. II. Eyes are the least important of the five senses. III. Eyes in humans are identical to eyes of other species used in animal models. IV. One eye can be treated while the other can act as an experimental control. a. I and II only b. II and III only c. III and IV only d. I and IV only https://www.hhmi.org/biointeractive/genes-medicine Genes as Medicine Genetics Published January 2018 www.BioInteractive.org Page 2 of 2 Short Film Student Handout 6. The gene therapy technique designed by Drs. Bennett and Maguire to cure Molly’s blindness involved using a virus as a vector to deliver the corrective gene into one of her eyes. What necessary step must be taken before viruses are used in this way? _______ a. Patients must first take flu medicine to help them avoid any flulike symptoms that the virus causes. b. The virus’s harmful genes and the ones the virus needs to replicate must be removed. c. Patients like Molly must first receive a vaccine for the virus so that they don’t get the disease that the virus carries. d. Doctors must first snip out the defective gene from a patient’s photoreceptor cells before using the virus to deliver the corrective gene. 7. Explain unique characteristics of viruses that make them useful tools for gene therapy. 8. As a final test of their technique to cure Leber amaurosis, Drs. Bennett and Magu.

1. Genetics Published January 2018
www.BioInteractive.org Page 1 of 2
Short Film
Student Handout
Genes as Medicine
NAME_______________________ DATE_________________
This handout supplements the short film Genes as Medicine.
1. True/False. Soon after a gene associated with childhood
blindness was discovered in the 1990s, Drs. Bennett
and Maguire were able to offer a gene therapy treatment for
children. _______
2. Explain the reasoning or evidence you used to answer
question 1.
3. In the film you met Molly, who has a form of childhood
blindness called Leber amaurosis (also called Leber
congenital amaurosis, or LCA). Her blindness is caused by
_______
a. early exposure to intensely bright light.
b. an eye injury she received at a young age.
c. mutations in a gene that is necessary for maintaining sight.

2. d. a viral disease in her nervous system that moved into her
eyes.
4. For many genetic diseases, children inherit the disease-
causing mutations from their parents. What was the
likely inheritance pattern in the case of Molly’s form of
childhood blindness? _______
a. Only females can have Leber amaurosis, so Molly must have
inherited the genetic mutation only from
her mother.
b. Molly became blind from new mutations that occurred in her
eye as a baby.
c. Molly ended up with childhood blindness because both of her
parents were blind when they were
children.
d. Molly inherited a mutation in the same gene from each of her
parents.
5. Which statements are reasons that eyes are a good target for
clinical trials related to genetic medicine?
_______
I. Eyes are easy to access.
II. Eyes are the least important of the five senses.
III. Eyes in humans are identical to eyes of other species used in

3. animal models.
IV. One eye can be treated while the other can act as an
experimental control.
a. I and II only
b. II and III only
c. III and IV only
d. I and IV only
https://www.hhmi.org/biointeractive/genes-medicine
Genes as Medicine
Genetics Published January 2018
www.BioInteractive.org Page 2 of 2
Short Film
Student Handout
6. The gene therapy technique designed by Drs. Bennett and
Maguire to cure Molly’s blindness involved using
a virus as a vector to deliver the corrective gene into one of her
eyes. What necessary step must be taken
before viruses are used in this way? _______
a. Patients must first take flu medicine to help them avoid any
flulike symptoms that the virus causes.
b. The virus’s harmful genes and the ones the virus needs to

4. replicate must be removed.
c. Patients like Molly must first receive a vaccine for the virus
so that they don’t get the disease that the
virus carries.
d. Doctors must first snip out the defective gene from a
patient’s photoreceptor cells before using the virus
to deliver the corrective gene.
7. Explain unique characteristics of viruses that make them
useful tools for gene therapy.
8. As a final test of their technique to cure Leber amaurosis,
Drs. Bennett and Maguire needed a large animal
model that was similar to humans.
a. What animal did they choose?
b. List at least three reasons that this animal was a good model
for testing a cure for childhood blindness.
9. Mutations to the RPE65 gene can cause Leber amaurosis.
Why is it a mistake to call RPE65 “the Leber
amaurosis gene”?
10. Sketch and annotate a diagram that shows how Molly ended
up inheriting non-functional alleles for the
gene associated with Leber amaurosis (Molly’s form of
childhood blindness).

5. NAME: DATE: question 1: question 3: question 4: question 5:
question 6: question 2: question 10: question 9: question 8b:
question 8a: Genetics: question 7:
Tracking Genetically Modified Mosquitoes
Biotechnology Revised July 2019
www.BioInteractive.org Page 1 of 3
Activity
Student Handout
INTRODUCTION
This activity accompanies the BioInteractive video Genetically
Modified Mosquitoes. In this activity, you will
provide questions and explore experiments to examine how
releasing genetically modified (GM) mosquitoes
impacts wild mosquitoes.
MATERIALS
• access to the video
• “Does Using GM Mosquitoes Work?” and “Data Tables”
handouts
• calculator and/or spreadsheet software
PRE-ACTIVITY
1. Describe your knowledge of or experience with mosquitoes.
2. For what diseases, in humans or other organisms, do

6. mosquitoes act as vectors (carriers)?
3. Watch the video Genetically Modified Mosquitoes and record
any questions you have. Briefly discuss the
video and your questions with a small group of other students.
PART 1: Research Questions
Imagine you and your family have recently moved to a small
town on the islands known as Key West off the
most southern tip of Florida. Life is good among the white
sandy beaches.
Health officials in town are growing concerned, however. News
reports from South America suggest that an
unusually large number of infants are being born with
abnormally small heads, a condition called microcephaly.
Health officials have identified the mosquito species Aedes
aegypti as a vector for the Zika virus that seems to be
causing this outbreak. Mosquitoes of this same species have
been found living in the Florida Keys. Alarmed city
officials want to do everything they can to prevent Zika virus
from spreading in Florida.
Some towns in Brazil have used genetically modified (GM)
mosquitoes to reduce the size of local mosquito
populations, as shown in the video Genetically Modified
Mosquitoes. Now officials in Florida are considering
releasing GM Ae. aegypti mosquitoes in your town. But first
they want to know if the method works. Let’s look
at the evidence.
4. Work with your group to develop one or two research
questions that would help determine whether
releasing GM mosquitoes into the environment is an effective
method for reducing wild mosquito
populations in your area. For each research question you

7. develop, fill out a table like the following.
http://www.hhmi.org/biointeractive/genetically-modified-
mosquitoes
http://www.hhmi.org/biointeractive/genetically-modified-
mosquitoes
Tracking Genetically Modified Mosquitoes
Biotechnology Revised July 2019
www.BioInteractive.org Page 2 of 3
Activity
Student Handout
Research question:
Data needed to answer the question:
Brief experimental design for collecting the data:
Predictions for your experiment: (what would the data show if
GM mosquitoes are effective in reducing wild mosquito
populations?)
5. Read through the handout “Does Using GM Mosquitoes
Work?” Compare and contrast the question,
experimental design, and the data described in the reading to
your research ideas.
PART 2: Data Analysis
6. Scientists at the company Oxitec completed an experiment in

8. Brazil similar to the one described in the “Does
Using GM Mosquitoes Work?” reading. Some of their data are
provided in the “Data Tables” handout. In this
same handout, fill in the missing rows for the “Brazilian Data:
Untreated Area” and “Brazilian Data: Treated
Area” data tables. These rows should show the number of traps
with eggs (L), the total number of traps (T),
the total number of eggs in all traps (E), the ovitrap index (OI),
and average density (AD).
7. On a separate piece of paper, create a line graph by plotting
the months on the x-axis and AD on the y-axis
for both untreated and treated areas. Place both untreated and
treated data on the same graph.
8. Using the information in the E and T rows of the data tables,
calculate the average AD, for the untreated and
treated areas, both before and after the mosquitoes were
released (Ub, Tb, Ua, and Ta). Then use these values
to compute the relative change in mosquito density in the
untreated versus treated areas.
For example, the average AD for the treated areas before
mosquitos were released is:
Tb = (6 + 30 + 42 + 59) eggs/(10 + 9 + 10 + 8) traps = 3.70
Use similar calculations, and the formula for relative change
shown below, to complete the following table.
Tb Ub Ta Ua relative change =
���������
�
���������

9. �
− ��
3.70
Tracking Genetically Modified Mosquitoes
Biotechnology Revised July 2019
www.BioInteractive.org Page 3 of 3
Activity
Student Handout
9. Use the data and evidence you gathered to make a claim
about whether the GM mosquito program is
effective in Brazil. Make sure to cite specific evidence to
support your claim.
10. Although there may be a difference in the average density of
mosquitoes between the untreated and
treated areas, there may be alternative explanations for the
difference other than the GM mosquito
program. To further investigate the impact of the GM mosquito
program, you will now calculate the fraction
of female mosquitoes mating with GM males in the wild.
a. Compute the mating fraction (M) for the missing months in
the “Mating Fraction Data: Treated Area”
table of the “Data Tables” handout.
b. On a separate piece of paper, create a bar graph by plotting

10. the month on the x-axis and the mating
fraction on the y-axis for the treated area.
c. Explain how the mating fraction evidence affects the claim
you made in Step 9.
11. Write a short letter to city officials summarizing evidence
(based on the Oxitec data) about whether
releasing GM mosquitoes may work in your area. Be sure to
emphasize the ultimate goal of the research and
the GM mosquito technique. Also describe any further questions
or concerns you have about the release.
PART 3: Additional Questions
12. Both the ovitrap index (OI) and the average density (AD)
are measures of the population dynamics of Ae.
aegypti. What ideas do you have for why the researchers use
two different measures?
13. Why was it valuable for researchers to include an untreated
area?
14. What additional data do you think the scientists might have
or could have collected at each study site on
factors that could influence the population dynamics of the
mosquitoes?
Tracking Genetically Modified
MosquitoesIntroductionMATERIALSPRE-ACTIVITYPART 1:
Research QuestionsPART 2: Data AnalysisPART 3: Additional
Questions
Research question: Data needed to answer the question: Brief
experimental design for collecting the data: question 1: question
2: Predictions for your experiment: question 3: u b: t a: u a:
relative change: question 5: question 12: question 14: question
13: question 10c: question 9:

11. RADIOLAB Podcast CRISPR
Listen to the Radiolab Podcast, “Antibodies Part 1: CRISPR.”
Click on the link to access the podcast. Then completely answer
all questions below.
Podcast URL
https://www.wnycstudios.org/story/update-crispr
Pre-listening discussion questions:
If you could custom order a “designer” dog or cat of any
realistic size, color, fur type, eye color, temperament, health
and intelligence, for an extra cost, would you choose to do so?
Explain your reasoning.
If scientists could do the same for unborn human babies, should
they do so? Should scientists have limits as to what traits could
be altered? Explain your reasoning.
Begin the podcast now.
1. Describe the strange thing that scientists found in
E. coli’s bacterial genome.
2. What does the acronym CRISPR stand for?

12. 3. What was the first big clue as to the function of these
repeating patterns?
4. What did Eugene Koonin suspect these sequences were for?
5. What is a marine bacterium’s greatest enemy? How bad is the
problem?
6. What is the role of enzymes as “ground troops” in fighting
this enemy? How successful are they?
7. If the enzyme ground troops win, what three things do the
bacterium do next?

13. 8. How do the molecular assassins use the RNA “mugshot “ to
target the enemy?
9. How did the “Dude” envision using this as a tool for
geneticists?
10. How does the new “good” gene get into place?
Pause at 16:00
Imagine being Jennifer Doudna……
How would you feel when you realized what this tool had the
potential to do?
After the discovery, how would you feel about the ethical issues
surrounding this potential? Do you feel any level of
responsibility or obligation to control its use?
Press play

14. 11. How is this gene editing tool better and more powerful than
those that came before it, according to Beth Shapiro?
12. How might extinct animals be brought back to life?
13. How could cancer be treated using CRISPR?
Stop at 20:00
Imagine 3 ways this technology could be used for positive
outcomes.
Now, imagine 3 ways this technology could be used for negative
outcomes.
Do you think the potential for positives outweighs the
negatives? Explain your reasoning.
Press play

15. 14. Where does Jennifer Doudna (“The Dude”) think we should
draw the line in using this technology, at least for now? Why?
15. What happened in China that has caused scientists to sound
the alarm?
Pause at 27:55
Did the Chinese scientist go too far in your opinion? Explain
your reasoning.
Press Play
16. Why isn’t CRISPR considered to be ready for use on human
embryos?
17. What diseases are being researched as potential target

16. 18. What is the East Coast-West Coast battle?
Stop at 38:23
Do you think someone should hold a patent on CRISPR, so that
no one else could use it without paying the patent holder?
Does the fact that CRISPR was found in nature and wasn’t
created by anyone, matter?
Press play.
19. How is CRISPR a potential alternative to antibiotics?
20. What is a gene drive system and how would it be used to
fight malaria?
21. What undesirable species are being targeted with gene drive
systems?

17. 22. What are some of the dangers that scientists imagine could
happen? What could go wrong?
Stop at 47:30 (end of podcast)
Is gene drive ethical to use in non-human wild species? Why or
why not? Explain your reasoning.
Is gene drive ethical to use in humans? Why or why not?
Explain your reasoning.
How do you deal with being unable to ask future generations
permission to alter their genes?

18. If you could, would you have your unborn child’s genes edited
with CRISPR (assuming it works as intended) to be immune to
Alzheimer's disease? Why or why not? Explain your reasoning.
What about editing your child’s genes to increase their
intelligence? Or eye color? Would you do that? Explain your
reasoning.
Part C: Biotechnology
The latter half of the twentieth century began with the discovery
of the structure of DNA, then progressed to the development of
the basic tools used to study and manipulate DNA. These
advances, as well as advances in our understanding of and
ability to manipulate cells, have led some to refer to the twenty-
first century as the biotechnology century. The rate of discovery
and of the development of new applications in medicine,
agriculture, and energy is expected to accelerate, bringing huge

19. benefits to humankind and perhaps also significant risks. Many
of these developments are expected to raise significant ethical
and social questions that human societies have not yet had to
consider.
In this section,
you can choose which biotechnology tool you'd like to
learn more about. In this week's discussion you'll make an
argument for or against the use of this technology.
Option 1: Genes as Medicine
Gene therapy is a genetic engineering technique that may one
day be used to cure certain genetic diseases. In its simplest
form, it involves the introduction of a non-mutated gene at a
random location in the genome to cure a disease by replacing a
protein that may be absent in these individuals because of a
genetic mutation. The non-mutated gene is usually introduced
into diseased cells as part of a vector transmitted by a virus,
such as an adenovirus, that can infect the host cell and deliver
the foreign DNA into the genome of the targeted cell. To date,
gene therapies have been primarily experimental procedures in
humans. A few of these experimental treatments have been
successful, but the methods may be important in the future as
the factors limiting its success are resolved.
For this option, you will watch the film below, and work
through this
worksheetIT IS GOING TO BE A PDF FILE CALLED
GENES OF MEDICINEFILL IT OUT about gene therapy and
its potential uses. You can answer the questions in the PDF
https://www.youtube.com/watch?v=GGEiaDau7hU&t=7s
Option 2: GM Mosquitoes
The
US EPA has approved an experimental permitLinks to
an external site. to have genetically modified mosquitoes

20. released in the Florida Keys. The GM mosquitoes have been
altered with a
gene driveLinks to an external site. which prevents
female mosquitoes from reaching maturity. The female
mosquitoes are the ones which bite, and potentially spread
deadly diseases.
For this option, you will watch the film below and complete
this
worksheetCALLED TRACKING GM MOSQUITOS IT
WILL BE A PDF FILE Only complete Part 1 of the worksheet.
Option 3: CRISPR-Cas9
CRISPR-Cas9 is a powerful gene editing tool that was
discovered in the bacteria
E. coli in the 1980s. CRISPR-Cas9 allows for the
removal, editing, or adding of DNA in precise locations.
CRISPR-Cas9 is a promising technology that could lead to
many breakthroughs in biotechnology and gene editing, though
it is not without controversy.
Here's a video summary of how CRISPR-Cas9 works:
https://www.youtube.com/watch?v=2pp17E4E-O8&t=2s
For this option, you will listen to the podcast episode below, or
download the episode here (mp3). To read the
transcript, visit the SITE
https://www.wnycstudios.org/podcasts/radiolab/articles/update-
crispr While listening to the episode, follow along
AND ANSWER QUESTIONS ON THE DOCUMENT
CALLED RADIOLAB Podcast CRISPR

21. PART D
Lab 7 Discussion: Should we or shouldn't we?
In this we investigated different types of biotechnology. Each
of these technologies holds the promise of tremendously
powerful ways of changing our world. The question is, should
we be using these technologies?
What you need to do
Describe briefly, in 2-3 sentences, how the technology you
investigated in Part C works and some examples of how the
technology is used.
Next, for the biotechnology you investigated in Part C of this
lab, construct an argument (5-7 sentences, minimum) that
expresses your opinion on whether these technologies should be
utilized or not. Some things to consider while thinking and
writing (these are guiding questions and not meant to be
answered individually):
· If this technology were widely used, who would be able to use
it?
· Who would benefit from the use of this technology?
· What are potential downsides (cons) of this technology?
· What are the upsides (pros) of this technology?

22. · What responsibility do scientist have to the safety and well
being of society?
· What is your mother/father/sibling/best friend's life could be
saved by using this technology - what would you tell them?
Due Date & Grading
Initial post must be between 7-10 sentences in length, minimum
image1.png