Rock, Paper, CRISPR!: The future of using gene editing technology to treat
human disorders and diseases
Abstract:
Designed to be completed at the end of a high school biology genetics unit (students must have
prior background knowledge of DNA, RNA, and the central dogma of molecular biology) this
gene editing project will introduce students to CRISPR-Cas9 gene editing technology and give
them an opportunity to reflect on the bioethics of editing human genomes. The project is split up
into 3 parts, the first part consisting of a self-exploration/research activity that will teach students
the history, critical components, and possible future applications of CRISPR with sources
carefully selected to be easily understood by high school students. For the second part of the
project, students will select and research a disorder or disease that could potentially be cured
using CRISPR-Cas9 technology, and design an informative poster to teach the class about their
disorder/disease and how CRISPR could be utilized to treat the disease. To conclude the
project, students will be given an opportunity to reflect on the risks and benefits of using
CRISPR-Cas9 to edit human genomes by writing a paragraph detailing their own opinion on
whether or not CRISPR-Cas9 should be utilized to edit human DNA.
Part 1: CRISPR self exploration questions:
Utilize the following links to answer the questions below about CRISPR-Cas9 technology:
Everything you need to know about CRISPR gene editing
What is CRISPR-Cas9?
CRISPR basics
Questions and answers about CRISPR
Components of CRISPR
HDR andNHEJ in CRISPR
Off target mutations and CRISPR bioethics
CRISPR mechanism image
1. How is CRISPR utilized by bacteria in nature?
2. What are the critical components of the CRISPR-Cas9 system?
CRISPRsCas9-
Guide RNA (sgRNA)-
crRNAtracrRNAProtospacer PAM-
3. What is the significance of Non-Homologous end joining and homology directed
repair with respect to the use of CRISPR-Cas9 technology:
4. How can the CRISPR system be optimized to possibly treat genetic diseases or
disorders?
5. What is an off-target effect and what is the significance of these off target effects
when considering the use of CRISPR in both germline and somatic cells?
6. What are some of the future implications of CRISPR-Cas 9 technology?
Part 2: Poster: Curing a human disease or disorder with CRISPR!
Poster time! For the next portion of our project, you will do research on a specific genetic or
other disease that could potentially be cured with the use of CRISPR-Cas9 technology, and then
create an informative poster describing the cause and symptoms of this disease, current
treatments, and how this disease could potentially be cured using the CRISPR-Cas9 system.
Below I’ve linked informative articles which discuss several genetic diseases and other
disorders that scientists have attempted to treat using the CRISPR-Cas9 system in animal or
in-vivo models, and in some cases, human models! any of these diseases would be a great
candidate for your project:
Treating diseases with CRISPR
Eight Diseases CRISPR technology could cure
You may also select a disease which is not listed in the links above, although it must be
approved by me first.
Poster Directions: You are to create an informative poster to present to the class that
includes the following criteria:
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●
●
●
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What is your disease or disorder?- include this in the title, try to make your title
unique!
What are the symptoms and cause of your disease?
What is the current prognosis and treatment options for your disease?
How can/is CRISPR-Cas9 gene editing technology being used to treat this
disease? (include illustrations of the CRISPR-Cas9 mechanism including role of
sgRNA and Cas9 as well as DNA repair).
Your poster can be structured however you like, but it must be colorful, neat,
scientifically accurate and must include a mix of writing and illustrations.
You may use any internet resources to gather information about the symptoms, cause,
and current treatment options for your selected disease, but make sure that it is
scientifically accurate!
Your finished poster will be completed on 22” x 28” poster board, but you can use the
boxes below to take notes that will aid you in completing your final poster:
Your disorder/disease:
Symptoms and cause of the disease:
Current prognosis and treatment:
How can the CRISPR/Cas9 system be
utilized in the future to possibly
treat/cure this disease in humans?
(what gene/genes would need to be
targeted by Cas9? Remember to include
a sketch and explanation of the basic
CRISPR-Cas9 mechanism in your
poster).
Part 3: What do you think? The bioethics of CRISPR-Cas9 in humans:
The use of CRISPR-Cas 9 to treat human disorders and diseases is a controversial topic in
bioethics due to the possible negative long term effects of using CRISPR to edit human
genomes. If germline cells are edited, the effects of CRISPR could last for several generations.
For the last portion of our CRISPR-Cas9 project, you will write a paragraph defending your own
opinion of using CRISPR to treat diseases and disorders in humans. Your paragraph must
address the following points:
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●
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What are the potential benefits of using CRISPR to treat human diseases?
What are some of the potential risks?
Do you personally believe the benefits outweigh the risks?
Should CRISPR-Cas9 gene editing technology be authorized for use in humans,
and if so, should parents be allowed to elect that this gene editing technology be
used on their children?
You can refer back to this CRISPR bioethics article to help you develop your paragraph:
Off target mutations and CRISPR bioethics
You may use the lines below to write your paragraph:
Part 1 answer key:
1. How is CRISPR utilized by bacteria in nature?
In nature, CRISPR is essentially an immune system used by bacteria to defend
against viruses that infect bacteria (bacteriophages). Some bacteria developed DNA
cutting proteins to chop up viral invaders. Snippets of the viral DNA are then
incorporated into the bacteria’s own genome so that they will remember the virus if it
invades again- these are the CRISPRs. If the same bacteriophage invades again, DNA
cutting proteins like Cas-9 are deployed to chop up and inactivate the viral invader.
2. What are the critical components of the CRISPR-Cas9 system?
CRISPRs- Clustered regularly interspaced short palindromic repeats
Cas9- protein component of the CRISPR-Cas9 sequence that is able to make cuts
in DNA sequences (these are like the CRISPR “scissors”).
Guide RNA (sgRNA)- A fusion between a crRNA and tracrRNA that is generated to
simplify experiments targeting a genomic target in experiments.
crRNA- small RNAs that contain a guide sequence that is complementary to a
sequence in bacteriophage DNA and a tag
tracrRNA- Binds to crRNA to help form sgRNA, a functional guide that Cas9
protein recognizes.
Protospacer - A sequence in viral DNA that pairs with the guide region of a crRNA.
The target of the CRISPR/Cas9 system.
PAM- A short sequence that is located next to the sequence targeted in viral DNA
but is NOT present in the CRISPR array
3. What is the significance of Non-Homologous end joining and homology directed
repair with respect to the use of CRISPR-Cas9 technology:
These are the two ways in which DNA can repair itself after a cut is made with
CRISPR. These repair mechanisms can help to induce insertion and deletion mutations
as well as introduce desired genes into a genome. NHEJ does not require a template to
guide repair, whereas HDR does. NHEJ is more active in cells and is more susceptible to
mutation errors due to Indels. HDR has a lower efficiency due to higher sequence
similarity needed between the severed and donor strands of DNA, but is less prone to
mutations. For this reason, HDR repaired DNA is usually more desirable when using
CRISPR for more controlled modifications.
4. How can the CRISPR system be optimized to possibly treat genetic diseases or
disorders?
CRISPR Cas9 technology can be used by scientists to induce mutations in a
gene responsible for causing a disease, rendering it non-functional, or it may also be
used to insert a functional gene into the genome to replace a mutated or non-functional
version. An sgRNA specific to the target gene is designed to guide Cas-9 to the correct
place in the genome to make a cut. This could lead to treating diseases like Huntington’s
and Sickle Cell Anemia, success in treating genetic disorders with CRISPR has already
been demonstrated in some animal models.
5. What is an off-target effect and what is the significance of these off target effects
when considering the use of CRISPR in both germline and somatic cells?
An off-target effect is when CRISPR-Cas9 technology induces a mutation or edits
DNA in an undesired location. This can be dangerous because off target effects can end
up causing more harm than good to an individual if a mutation is accidently induced in a
gene whose function is vital to proper health and survival. If off target mutations occur in
somatic cells, they may harm the individual undergoing CRISPR therapy, however, if they
occur in germline (sex) cells, these unwanted mutations may be passed on for
generations. For this reason, using CRISPR-Cas9 technology to edit human genomes is a
highly controversial topic in bioethics.
6. What are some of the future implications of CRISPR-Cas 9 technology?
In the future, CRISPR technology may be used to cure genetic diseases like
Cystic fibrosis, sickle cell anemia, Huntington’s disease, and several others. CRISPR may
also be used to treat certain cancers and viral diseases like COVID-19. If ever made
widely available and affordable for use in humans, the technology may even someday be
used to create desirable phenotypes in unborn babies.
Rubric:
Point Scale:
Part 1: CRISPR
knowledge questions
25
All questions answered
correctly
Part 2: Informative
poster
-
-
-
Name of disease
and title included
Symptoms and
cause of disease
included
Current
prognosis and
treatment
included
CRISPR
information and
illustration
included
Poster is neat,
colorful, and
includes a mix of
writing and
pictures
Part 3: Bioethics
paragraph
All 4 required criteria are
addressed and the
paragraph is very
thoughtful (risks/benefits,
personal beliefs, should
CRISPR be authorized for
use in humans?).
20
5 questions answered
correctly
Missing 1 of the required
criteria
3 required criteria
addressed or all required
criteria addressed but
paragraph is somewhat
lacking in critical
thinking/substance.
15
4 questions answered
correctly
Missing 2 of the required
criteria
2 required criteria
addressed or 3-4 required
criteria addressed but
paragraph is clearly
lacking in critical
thinking/substance
10
3 questions answered
correctly
Missing 3 of the required
criteria
1-2 required criteria
addressed and paragraph
is severely lacking in
critical thinking/substance
5
1-2 questions answered
correctly
Missing 4 of the required
criteria
0-1 required criteria
addressed and paragraph
is severely lacking in
critical thinking/substance
0
Missing or all questions
incorrect
Missing all required
criteria or poster is
illegible with very little
effort put into it.
Missing