CRISPR – Gene Editing Assignment
Information/History of CRISPR Gene Editing Technology:
CRISPR/Cas9 is a simple but powerful gene-editing technology that we can harness to
precisely modify, delete or correct disease-causing abnormalities at their genetic sources.
“CRISPR” refers to Clustered Regularly Interspaced Short Palindromic Repeats of genetic
information. CRISPR-Cas9 as a genome-editing tool
The genomes of various organisms encode a series of messages and instructions within their DNA
sequences. Genome editing involves changing those sequences, thereby changing the messages.
This can be done by inserting a cut or break in the DNA and tricking a cell's natural DNA repair
mechanisms into introducing the changes one wants. CRISPR-Cas9 provides a means to do so.
"Operationally, you design a stretch of 20 [nucleotide] base pairs that match a gene that you want to
edit," said George Church, Robert Winthrop Professor of Genetics at Harvard Medical School. An
RNA molecule complementary to those 20 base pairs is constructed. Church emphasized the
importance of making sure that the nucleotide sequence is found only in the target gene and
nowhere else in the genome. "Then the RNA plus the protein [Cas9] will cut — like a pair of scissors
— the DNA at that site, and ideally nowhere else," he explained.
Once the DNA is cut, the cell's natural repair mechanisms kick in and work to introduce mutations or
other changes to the genome. There are two ways this can happen. According to the Huntington's
Outreach Project at Stanford (University), one repair method involves gluing the two cuts back
together. This method, known as "non-homologous end joining," tends to introduce errors.
Nucleotides are accidentally inserted or deleted, resulting in mutations, which could disrupt a gene.
In the second method, the break is fixed by filling in the gap with a sequence of nucleotides. In order
to do so, the cell uses a short strand of DNA as a template. Scientists can supply the DNA template
of their choosing, thereby writing-in any gene they want, or correcting a mutation.
CRISPR/Cas9 gene editing has transformed many areas of research. Thousands of academic
laboratories across the world have adopted CRISPR/Cas9, using the technology to conduct a wide
range of new experiments. The rapid adoption of CRISPR/Cas9 and the resulting groundbreaking
research have driven tremendous progress in the field, informing our work and others’.
CRISPR/Cas9 To Treat Diseases
There are more than 10,000 known single-gene (or monogenic) diseases, occurring in about 1 out of
every 100 births1. Scientists and clinicians are now conducting pioneering research using
CRISPR/Cas9 to address both recessive and dominant genetic defects, opening up the potential of
gene editing to provide novel transformative gene-based medicines for patients with a large number
of both rare and common diseases.
We have established a portfolio of programs by selecting disease targets based on a number of
criteria, including high unmet medical need, advantages of CRISPR/Cas9 relative to alternative
approaches, technical feasibility and the time required to advance the product candidate into and
through clinical trials. For CRISPR/Cas9-based therapeutics, technical feasibility is primarily
determined by the delivery modality and by the editing strategy required to treat the disease.
Our Therapeutic Approach
CRISPR Therapeutics’ mission is to develop transformative gene-based medicines that offer the
possibility of curative treatments. Our therapeutic approach is to cure diseases at the molecular level
using CRISPR/Cas9 gene editing.
Our portfolio of therapeutic candidates spans three main categories: ex vivo treatments for
hemoglobinopathies, such as sickle cell disease and β-thalassemia, allogeneic CAR-T cell therapies
for cancer, and in vivo treatments for diseases of the liver, lung, and muscle.
We are focused on the treatment of somatic (non-germline) cells – cells that are not heritable. We
are not using human germline modifications and we support the current recommendations of
the International Society for Stem Cell Research in this regard. Together with other gene-editing
companies, we have issued a Joint Statement - Position Regarding Human Germline Gene Editing.
Our development of transformative new medicines will involve working closely with patients and
families, healthcare professionals, regulatory agencies and other groups dedicated to improving
healthcare.
YOUR ASSIGNMENT:
With GENE Editing on the Horizon you have a family member or maybe you yourself
(hypothetically) have a genetic mutation that has caused a disease. Your job is to research all about this
new therapy. It’s the Buzz word in the world of Medical and Biotech….CRISPR.
In a presentation, please present the following to “us” your class as if you are a scientist presenting the
information and the future implications of this new found therapy. You must answer all questions on the
Google Doc SLIDES. You will be presenting this information to the class on Friday, February 23rd. The
presentation must be emailed to me faith.schumann@apps.mca.lwcc.org
1. In your table Group…(no switching groups) choose a genetic disease caused from a mutation to
present on and ask me if that disease will work for your group.
2. Describe in detail:
-What the disease is called?
- What are the symptoms of the disease?
- On what chromosome number is this disease mutation found?
- What is the name of the gene called that this mutation is found?
- Name the number of people affected by this disease/year in the US
3. How would CRISPR gene therapy work to correct this mutation, find a way to show how this fix
would occur. Pictures or Videos allowed.
4. What makes CRISPR therapy so unique and pertain to what we are learning in Biology Class? Use
key words, DNA, RNA, primers, enzymes, etc.
5. Does CRISPR gene editing totally repair the mutations and/or how long will it last?
6. Would the gene editing be passed down to generations? In other words will this therapy allow
for offspring to not get the same genetic disease?
7. What are the Ethical concerns with this new therapy, PLEASE present 2-3 concerns.
8. Are there any drawbacks to this therapy?
9. What are the costs to this therapy?
10. Considering your disease and the ethical implications, would you take this therapy if it meant to
have your disease be “corrected”?