Module 3 Activity # 4

Name: JANINE A. POLISON Date: 02/03/2023

Course/Yr. & Sec.: MM 1-A

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Self-Check Test

A. Question and Answer: Answer the questions briefly but correctly. Write your
answers below each number.

1. How is gene therapy characterized?

Answer: That Involves inserting one or more connective genes that have been
designed in the laboratory, into the genetic material of a patient’s cells to cure a
genetic disease.

2. What can be altered by the expression of the new gene or genes to correct the
disease?

Answer: Altered the DNA or RNA transcript used in the synthesis of proteins

3. In the gene therapy using an adenovirus vector, a new gene is injected into an
adenovirus vector. What is the use of the adenovirus vector?

Answer: Introduce the modified DNA into a human cell

4. What happens to the new gene if the treatment is successful?

Answer: The new gene will make a functional protein.

5. In the future, the gene therapy technique may allow doctors to treat a disorder by
inserting a gene into a patient’s cells. What presently used medical technologies
can be replaced by gene therapy?

Answer: Using of drugs or surgery

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Module 3 Activity # 4

6. Although gene therapy is a promising treatment option for a number of diseases,

the technique remains risky and is still under study. Why?

Answer: Make sure that it will be safe and effective.

7. Gene therapy works by replacing a faulty disease-causing gene with a working

version, or by introducing a new gene to cure a condition or modify its effects.
What is its aim?

Answer: Eliminate genetic diseases at their source

8. With new scientific gene therapy improvements, the abnormal or absent genes
can be replaced with healthy ones. What does it enable cells to?

Answer: To produce useful proteins

9. Cell and gene therapies are revolutionizing cancer treatment. How?

Answer: Repairing, replacing, or destroying cancerous cells

10. When a cell does not work properly or is absent, a genetically engineered gene is
inserted into the cell. What is necessary for proper functioning of the cell?

Answer: The gene needs to be inserted correctly into the target cell to avoid
causing any adverse effects.

11. What is the challenge for nations experimenting with gene therapy?

Answer: To come up with workable, fair and ethical guidelines for its use

12. What is the target of gene therapy?

Answer: The targert the faulty genes responsible for genetic diseases

13. What can inheriting a faulty gene directly cause?

Answer: Wide range of disorders such as cystic fibrosis and haemophilia

14. What can gene therapy be used to?

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Module 3 Activity # 4

Answer: Used to replace a faulty gene with a healthy version or to introduce a

new gene that can cure a condition or modify its effects

15. Genes are the blueprint for our bodies. What do genes provide?

Answer: Information for the cells to produce proteins and enzymes to control
our growth, development and health

16. What does gene mutation mean?

Answer: Gene contains a variation or “spelling mistake” that disrupts the gene
message.

17. Sometimes, the whole or part of the gene is missing (deleted). These changes
can make the gene faulty. How can a mutation occur?

Answer: Spontaneously.

18. Genes are responsible for every aspect of cell life. What do genes hold?

Answer: Code for proteins that enable cells to grow, function and divide

19. When genes are not properly functioning or are missing or defective, what is the
goal of gene therapy?

Answer: The goal of gene therapy is to fix the problems at the source using cell
and gene therapy.

20. Generally, in gene therapy a gene cannot be directly inserted into a person’s cell.
How can a gene be delivered to the cell?

Answer: It must be delivered to the cell using a carrier, or vector

B. Identification: Identify what is referred to in each item. Write the letter of your
answer on the blank provided for each number.

Choices

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Module 3 Activity # 4

A. restores the target cell to a normal state

B. unloads its genetic material containing the therapeutic human gene into the
target cell
C. causing inflammation
D. These are carried on the sperm and egg cells
E. target other cells as well as the intended cells and may be contagious
F. To carry normal human DNA
G. These are immature cells that have the potential to develop into cells with
different functions.
H. treating acquired conditions such as cancer and AIDS
I. treat genetic diseases, viral diseases, and cancer
J. causing other health problems
K. eliminate genetic diseases at their source
L. delivering their genetic material into the host cell to create more of their virus in
the body
M. To come up with workable, fair and ethical guidelines for its use.
N. replacing a faulty disease-causing gene with a working version, or by introducing
a new gene to cure a condition or modify its effects
O. They may stitch.
P. mutation can occur spontaneously

F 1. Currently, the most common type of vectors are viruses that have been genetically
altered. What is the purpose of which?

B 2. Target cells such as the patient’s liver or lung cells are infected with the vector.
What is the function of the vector?

A 3. The therapeutic gene generates a functional protein product. What is the effect?

G 4. What are stem cells?

D 5. A replaced, working gene that is inserted into the cells in the body that are
affected would cure the individual. It would not prevent their children from inheriting the
original faulty gene. Why?

C 6. As a risk of gene therapy, the immune system may respond to the working gene
copy that has been inserted. How?

J 7. The working gene might produce too much of the missing enzyme or protein.
What is the effect?

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Module 3 Activity # 4

E 8. The risks of gene therapy are the possible results that might happen on the
following: a) working gene, it might be slotted into the wrong spot, b) other genes, may
be accidentally delivered to the cell, and in c) deactivated virus, what might happen?

H 9. The majority of gene therapy trials are being conducted in the US and Europe.
What do most trials focus on?

I 10. Gene editing is a potential approach to alter the human genome. What is its
purpose?

M 11. What is the challenge for nations experimenting with gene therapy?

N 12. How does a gene therapy work?

K 13. What is the aim of gene therapy?

L 14. How do viruses function in gene therapy?

O 15. What is the risk involved with retroviruses?

C. Fill-in-the Blank: Fill the blank with the correct and best answer by writing your
answer on the blank provided before each number.

Choices

A. that’s what viruses evolved to do with their own genetic material

B. immune response from the body, putting a patient at risk of further illness
C. it lacks the genes required to spread itself on its own
D. natural tendency of viruses to infect certain organs
E. replacing all of the defective cells
F. block the flow of blood
G. replace missing or malfunctioning genes
H. because blood-clotting factors can be added to the blood in the liver
I. only part of the brain must be targeted
J. the absence of adenosine deaminase
K. delivery of DNA into cells
L. lower therapeutic efficacy
M. sticks the gene into the human cells
N. human gene therapy
O. eliminate genetic diseases at their source

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Module 3 Activity # 4

P. genetic material into the host cell

G 1. Gene therapy is the addition of new genes to a patient’s cells in order to replace
missing or malfunctioning genes

A 2. Researchers in gene therapy use a virus to carry the genetic cargo into cells
because that’s what viruses evolved to do with their own genetic material

I 3. Researchers are trying to treat Parkinson’s disease because only part of the
brain must be targeted Other diseases can be treated in this approach such as eye
diseases and haemophilia.

B 4. Early in-the-body gene therapies used a virus called adenovirus-the virus behind
the common cold. Adenovirus when used in gene therapy can cause immune
response from the body, putting a patient at risk of further illness

C 5. Today, researchers use a virus called adeno-associated virus, which is not known
to cause any disease in humans. In nature, this agent needs to hitch a ride with an
adenovirus because it lacks the genes required to spread itself on its own.

D 6. In gene therapy, viruses are used as vectors because of the natural tendency of
viruses to infect certain organs

H 7. Adeno-associated virus goes straight for the liver when it is injected into the
bloodstream. Adeno-associated viruses are used in gene therapies to treat haemophilia
because blood-clotting factors can be added to the blood in the liver

E 8. In out-of-the-body gene therapy, researchers take blood or bone marrow from a

patient and separate out immature cells. They then add a gene to those cells and inject
them into the bloodstream of the patient. The cells travel to the bone marrow, mature
and multiply rapidly, eventually replacing all of the defective cells

F 9. Doctors are working on the ability to do out-of-the-body gene therapy to replace all
of a patient’s bone marrow or the entire blood system, as would be useful in sickle-cell
anemia. In sickle-cell anemia, the red blood cells are shaped like crescents. The effect

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Module 3 Activity # 4

of these red blood cells that are shaped like crescents cause them to block the flow of
blood

J 10. In the history of human gene therapy, severe combined immunodeficiency was
the first monogenic disorder for which gene therapy was developed. This severe
combined immunodeficiency is cause by the absence of adenosine deaminase

M 11. A virus is a vector. This means it is a carrier; the gene has been inserted into
the viral genome. The virus sticks the gene into the human cells

K 12. The delivery of DNA into cells can be accomplished by multiple methods. The
two major classes are recombinant viruses (viral vectors) and naked DNA or DNA
complexes (non-viral methods).

P 13. Viruses introduce their genetic material into the host cell tricking the host’s
cellular machinery into using it as blueprints for viral proteins in order to replicate.

N 14. A number of viruses have used for human gene therapy. These viruses are:
retrovirus; adenoviruses; herpes simplex, vaccinia, and adeno-associated virus.

L 15. Non-viral methods initially produced lower levels of transfection and gene
expression. The effect is lower therapeutic efficacy

Assessment for Learning

A. Identification: Identify the correct and best answer of the following. Choose
your answer from the choices provided. Write the letter of your answer on the
blank before each item.

Choices

A. produce useful proteins

B. DNA or RNA transcript used in the synthesis of proteins
C. The new gene will make a functional protein.
D. repairing, replacing, or destroying cancerous cells

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Module 3 Activity # 4

E. drugs or surgery
F. Involves inserting one or more connective genes that have been designed in
the laboratory, into the genetic material of a patient’s cells to cure a genetic
disease.
G. eliminate genetic diseases at their source
H. introduce the modified DNA into a human cell
I. The gene needs to be inserted correctly into the target cell to avoid causing
any adverse effects.
J. To come up with workable, fair and ethical guidelines for its use
K. replace a faulty gene with a healthy version or to introduce a new gene that
can cure a condition or modify its effects
L. a gene contains a variation or “spelling mistake” that disrupts the gene
message.
M. wide range of disorders such as cystic fibrosis and haemophilia
N. information for the cells to produce proteins and enzymes to control our
growth, development and health
O. faulty genes responsible for genetic diseases
P. spontaneously
Q. fix the problems at the source using cell and gene therapy
R. code for proteins that enable cells to grow, function and divide
S. carry normal human DNA
T. using a carrier, or vector
U. restores the target cell to a normal state
V. unloads its genetic material containing the therapeutic human gene into the
target cell
W. These are carried on the sperm and egg cells
X. immature cells that have the potential to develop into cells with different
functions.
Y. only part of the brain must be targeted
Z. Make sure that it will be safe and effective.

C 1. What happens to the new gene if the treatment is successful?

E 2. In the future, the gene therapy technique may allow doctors to treat a disorder by
inserting a gene into a patient’s cells. What presently used medical technologies can be
replaced by gene therapy?

A 3. With new scientific gene therapy improvements, the abnormal or absent genes
can be replaced with healthy ones. What does it enable cells to?

D 4. Cell and gene therapies are revolutionizing cancer treatment. How?

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Module 3 Activity # 4

F 5. How is gene therapy characterized?

B 6. What can be altered by the expression of the new gene or genes to correct the
disease?

H 7. In the gene therapy using an adenovirus vector, a new gene is injected into an
adenovirus vector. What is the use of the adenovirus vector?

O 8. What is the target of gene therapy?

M 9. What can inheriting a faulty gene directly cause?

Z 10. Although gene therapy is a promising treatment option for a number of diseases,
the technique remains risky and is still under study. Why?

G 11. Gene therapy works by replacing a faulty disease-causing gene with a working
version, or by introducing a new gene to cure a condition or modify its effects. What is
its aim?

I 12. When a cell does not work properly or is absent, a genetically engineered gene is
inserted into the cell. What is necessary for proper functioning of the cell?

J 13. What is the challenge for nations experimenting with gene therapy?

K 14. What can gene therapy be used to?

N 15. Genes are the blueprint for our bodies. What do genes provide?

L 16. What does gene mutation mean?

P 17. Sometimes, the whole or part of the gene is missing (deleted). These changes
can make the gene faulty. How can a mutation occur?

R 18. Genes are responsible for every aspect of cell life. What do genes hold?

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Module 3 Activity # 4

Q 19. When genes are not properly functioning or are missing or defective, what is the
goal of gene therapy?

T 20. Generally, in gene therapy a gene cannot be directly inserted into a person’s cell.
How can a gene be delivered to the cell?

S 21. Currently, the most common type of vectors are viruses that have been
genetically altered. What is the purpose of which?

V 22. Target cells such as the patient’s liver or lung cells are infected with the vector.
What is the function of the vector?

U 23. The therapeutic gene generates a functional protein product. What is the effect?

X 24. What are stem cells?

W 25. A replaced, working gene that is inserted into the cells in the body that are
affected would cure the individual. It would not prevent their children from inheriting the
original faulty gene. Why?

B. Fill-in-the Blank: Fill the blank with the correct and best answer by writing your
answer on the blank provided before each number.

Choices

A. that’s what viruses evolved to do with their own genetic material

B. immune response from the body, putting a patient at risk of further illness
C. it lacks the genes required to spread itself on its own
D. natural tendency of viruses to infect certain organs
E. replacing all of the defective cells
F. block the flow of blood
G. replace missing or malfunctioning genes
H. because blood-clotting factors can be added to the blood in the liver
I. only part of the brain must be targeted
J. the absence of adenosine deaminase
K. delivery of DNA into cells
L. lower therapeutic efficacy
M. sticks the gene into the human cells

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Module 3 Activity # 4

N. human gene therapy

O. eliminate genetic diseases at their source
P. genetic material into the host cell
Q. delivering their genetic material into the host cell to create more of their virus in
the body
R. causing inflammation
S. they may stitch
T. target other cells as well as the intended cells and may be contagious
U. treat genetic diseases, viral diseases, and cancer
V. eliminate genetic diseases at their source
W. come up with workable, fair and ethical guidelines for its use
X. replacing a faulty disease-causing gene with a working version, or by introducing
a new gene to cure a condition or modify its effects
Y. causing other health problems
Z. treating acquired conditions such as cancer and AIDS

G 1. Gene therapy is the addition of new genes to a patient’s cells in order to replace
missing or malfunctioning genes

A 2. Researchers in gene therapy use a virus to carry the genetic cargo into cells
because that’s what viruses evolved to do with their own genetic material

I 3. Researchers are trying to treat Parkinson’s disease because only part of the
brain must be targeted Other diseases can be treated in this approach such as eye
diseases and haemophilia.

B 4. Early in-the-body gene therapies used a virus called adenovirus-the virus behind
the common cold. Adenovirus when used in gene therapy can cause immune
response from the body, putting a patient at risk of further illness

C 5. Today, researchers use a virus called adeno-associated virus, which is not known
to cause any disease in humans. In nature, this agent needs to hitch a ride with an
adenovirus because it lacks the genes required to spread itself on its own.

D 6. In gene therapy, viruses are used as vectors because of the natural tendency of
viruses to infect certain organs

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Module 3 Activity # 4

H 7. Adeno-associated virus goes straight for the liver when it is injected into the
bloodstream. Adeno-associated viruses are used in gene therapies to treat haemophilia
because blood-clotting factors can be added to the blood in the liver

E 8. In out-of-the-body gene therapy, researchers take blood or bone marrow from a

patient and separate out immature cells. They then add a gene to those cells and inject
them into the bloodstream of the patient. The cells travel to the bone marrow, mature
and multiply rapidly, eventually replacing all of the defective cells

F 9. Doctors are working on the ability to do out-of-the-body gene therapy to replace all
of a patient’s bone marrow or the entire blood system, as would be useful in sickle-cell
anemia. In sickle-cell anemia, the red blood cells are shaped like crescents. The effect
of these red blood cells that are shaped like crescents cause them to block the flow of
blood

J 10. In the history of human gene therapy, severe combined immunodeficiency was
the first monogenic disorder for which gene therapy was developed. This severe
combined immunodeficiency is cause by the absence of adenosine deaminase

M 11. A virus is a vector. This means it is a carrier; the gene has been inserted into
the viral genome. The virus sticks the gene into the human cells

K 12. The delivery of DNA into cells can be accomplished by multiple methods. The
two major classes are recombinant viruses (viral vectors) and naked DNA or DNA
complexes (non-viral methods).

P 13. Viruses introduce their genetic material into the host cell
tricking the host’s cellular machinery into using it as blueprints for viral proteins in order
to replicate.

N 14. A number of viruses have used for human gene therapy These viruses are:
retrovirus; adenoviruses; herpes simplex, vaccinia, and adeno-associated virus.

L 15. Non-viral methods initially produced lower levels of transfection and gene
expression. The effect is lower therapeutic efficacy

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Module 3 Activity # 4

Q 16. Viruses function in gene therapy delivering their genetic material into the
host cell to create more of their virus in the body

S 17. The risk involved with retroviruses is that they may stitch

R 18. As a risk of gene therapy, the immune system may respond to the working gene
copy that has been inserted by causing inflammation

Y 19. The working gene might produce too much of the missing enzyme or protein
causing other health problems

T 20. The risks of gene therapy are the possible results that might happen on the
following: a) working gene, it might be slotted into the wrong spot, b) other genes, may
be accidentally delivered to the cell, and in c) deactivated virus, target other cells as
well as the intended cells and may be contagious

Z 21. The majority of gene therapy trials are being conducted in the US and Europe.
Most trials focus on treating acquired conditions such as cancer and AIDS

U 22. Gene editing is a potential approach to alter the human genome. The purpose
is to treat genetic diseases, viral diseases, and cancer

W 23. the challenge for nations experimenting with gene therapy is to come up with
workable, fair and ethical guidelines for its use

X 24. A gene therapy work by replacing a faulty disease-causing gene with a

working version, or by introducing a new gene to cure a condition or modify its
effects

V 25. The aim of gene therapy is to eliminate genetic diseases at their source

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