GE1713 Topic Learnings

Name: ____________________________________ Section: ___________________ Date: _________

Instructions: Reflect on the topics that were previously discussed. Write at least three (3) things per
topic that you have learned. Answers must be written in complete sentences.
TOPIC LEARNINGS

Gene Therapy
(Definition and Principles)
Gene therapy is a technique that modifies a person's genes to treat or
cure disease. Gene therapies can work by several mechanisms: Replacing
a disease-causing gene with a healthy copy of the gene. Inactivating a
disease-causing gene that is not functioning properly.
Gene therapy involves the transfer of functional genes into somatic cells
and their expression in target tissues in order to replace absent genes,
correct defective genes, or induce antitumoral activity in the tumor-
bearing host.

Gene Editing Genome editing is a way of making specific changes to the DNA of a cell
or organism. An enzyme cuts the DNA at a specific sequence, and when
this is repaired by the cell, a change or ‘edit’ is made to the sequence.
• It is a technique used to precisely and efficiently modify DNA within a
cell.
• It involves making cuts at specific DNA sequences with enzymes called
‘engineered nucleases’. • It can be used to add, remove, or alter DNA in
the genome.

CRISPR-Cas9 CRISPR-Cas9
• CRISPR-Cas9 is the most common, cheap, and efficient system used for
genome editing. • CRISPR stands for ‘clustered regularly interspaced
short palindromic repeats’.
• CRISPR is the DNA-targeting part of the system which consists of an
RNA molecule, or ‘guide’, designed to bind to specific DNA bases through
complementary base-pairing.
• Cas9 stands for CRISPR-associated protein 9 and is the nuclease part
that cuts the DNA.
• The CRISPR-Cas9 system was originally discovered in bacteria that use
this system to destroy invading viruses.

10 Worksheet 1 *Property of STI Page 1 of 2

GE1713 Topic Learnings

Name: ____________________________________ Section: ___________________ Date: _________

Instructions: Reflect on the topics that were previously discussed. Write at least three (3) things per
 topic that you have learned. Answers must be written in complete sentences.
TOPIC
Important Events in
Gene Therapy
Diseases Being Studied
for Gene Therapy
Issues and Concerns
10 Worksheet 1 *Property of STI Page 2 of 2
LEARNINGS
The concept of human gene therapies emerged in 1989, with the first
approved human gene therapy trial of Rosenberg et al. This trial
investigated the use of human tumor-infiltrating lymphocytes (TIL)
genetically modified by introducing the gene coding for resistance to
neomycin into TIL for patients with advanced melanoma. TILs can
overcome tumors' heterogeneity and immune escape and provide better
clinical outcomes than chimeric antigen receptor (CAR)-T cells in treating
solid tumors with high mutation rates, such as melanoma .
Cancer was the most common indication for gene therapies in
development (64.41%), including different types of cancer:
gynecological; nervous system; gastrointestinal; genitourinary; skin; head
and neck; lung; mesothelioma; hematological; and sarcoma.The second
most popular indication for gene therapies was monogenic diseases; it
was targeted by 10% of all the gene therapy trials. This is not surprising,
as those diseases are related to one single gene defect and gene
therapies are potentially able to correct the gene defect.
Genetic therapies hold promise to treat many diseases, but they are still
new approaches to treatment and may have risks. Potential risks could
include certain types of cancer, allergic reactions, or damage to organs or
tissues if an injection is involved. Recent advances have made genetic
therapies much safer. Like the story of Woman’s Death Calls Gene
Therapy into Question, Why Gene Therapy Caused Leukemia in Some
“Boy in the Bubble Syndrome” Patients, FDA halts Gene Therapy Trials
After Leukemia Case in France, and Death but One Unintended
Consequence of Gene Therapy Trial