MODULE 13 166

Gene Therapy

HOW TO LEARN FROM THIS MODULE

Here’s a simple guide for you in going about the module:
1. Read and follow the instructions carefully.
2. Read each lesson and do all the activities provided for you.
3. Perform all the activities diligently to help and guide you in understanding the topic.
4. Take the Learning Activities after each lesson to determine how well you understood
the topic.
5. Answer the Assessment Task at the end of the module to measure how much you have
gained from the lessons. You may answer it via the online link provided or write your
answers on a sheet of paper that you can physically submit, or you may take a picture
of your answers and send it via messaging platforms with which you agreed with your
instructor during the class orientation.

INTRODUCTION

In the field of medicine, medical researchers are continuously looking for different
ways of treating genetic disorders and gene-related illnesses. With the advent of molecular
biology, the causes of genetic disorders were now being traced and defective genes are
now detected. A deeper understanding of the molecular components of the body such as
genes, their functions, locations and control mechanisms paved way for coming up of a
clearer technique in treating disorders. These ushered the development of treating genetic
disorders called gene therapy. The initial aim of gene therapy was the correction of
inherited genetic diseases.

After completing the module, you should be able to answer the question:
 What are potential benefits and issues of gene therapy on health?

LEARNING OBJECTIVES

At the end of this module, the learner should be able to:

o Describe gene therapy and its various forms;

o Discuss the benefits and risks of gene therapy; and
o Assess the issue’s potential benefits detriment to global health

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LEARNING CONTENT

Gene therapy is when a DNA is introduced into a patient to treat a genetic disease.
The new DNA contains a functioning gene to correct the effects of a disease causing
mutation. It is also an experimental technique that uses genes to treat or prevent disease.
This technique may allow doctors to treat a disorder by inserting a gene into a patient’s
cells instead of using drugs or surgery.

LESSON 1. How does gene therapy work?

Gene therapy is designed to introduce genetic material into cells to compensate for
abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary
protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the
gene to restore the function of the protein.

A gene that is inserted directly into a cell usually does not function. Instead, a carrier
called a vector is genetically engineered to deliver the gene. Certain viruses are often used
as vectors because they can deliver the new gene by infecting the cell. The viruses are
modified so they can't cause disease when used in people. Some types of virus, such as
retroviruses, integrate their genetic material (including the new gene) into a chromosome in
the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus
of the cell, but the DNA is not integrated into a chromosome.

The vector can be injected or given intravenously (by IV) directly into a specific
tissue in the body, where it is taken up by individual cells. Alternately, a sample of the
patient's cells can be removed and exposed to the vector in a laboratory setting. The cells
containing the vector are then returned to the patient. If the treatment is successful, the new
gene delivered by the vector will make a functioning protein.

Researchers must overcome many technical challenges before gene therapy will be a
practical approach to treating disease. For example, scientists must find better ways to
deliver genes and target them to particular cells. They must also ensure that new genes are
precisely controlled by the body.

A new gene is inserted directly into a cell. A carrier called a vector is genetically
engineered to deliver the gene. An adenovirus introduces the DNA into the nucleus of the
cell, but the DNA is not integrated into a chromosome.

Types of Gene Therapy

1. Somatic gene therapy – transfer of a section of DNA to any cell of the body that
doesn’t produce sperm or eggs.
2. Germline gene therapy – transfer of a section of DNA to cells that produces sperm or
eggs.

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Fig.13.1 Gene Therapy using an Adenovirus Vector

Source: https://ghr.nlm.nih.gov/primer/therapy/procedures

Categories of Gene Therapy:

1. Somatic-Cell gene therapy for the cure and prevention of diseases. Example :
insertion of a DNA sequence into a person’s cell to allow production of an enzyme
like adenosine deaminase.
2. Germ Line gene therapy for cure and prevention of diseases. Example: Insertion of
an adenosine deaminase sequence into early embryo or reproductive cells, which
would affect not only the individual but all of his or her offspring.
3. Somatic cell enhancement. Example: Insertion of a DNA sequence to improve
memory , increase height, or increase intelligence which would affect only the
individual.
4. Germ line enhancement. Example: Insertion of a DNA sequence for enhancement
into a blastocyst, sperm or egg, which would affect future generation.

Gene Therapy Techniques

There are several ways by which gene therapy is administered. This includes the
following:

1. Gene augmentation therapy. It is designed for diseases caused by loss of function of a

gene, introducing extra copies of a normal gene may increase the amount of normal gene
product to a level where the normal phenotype is restored.

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2.Gene inhibition therapy. This is suitable for the treatment of infectious diseases, cancer
and inherited diseases caused by the inappropriate gene activity. The aim is to introduce a
gene whose product either: inhibits the expression of another gene or interferes with the
activity of the product of another gene.

3. Gene therapy killing of specific cells. Genes are directed to the target cells and then
expressed so as to cause cell killing. Direct cell killing is possible if the inserted genes are
expressed to produce a lethal toxin(suicide gene) or a gene encoding a prodrug is inserted,
conferring susceptibility to killing by a subsequently administered drug.

Fig.13.2 Gene Therapy Techniques

Source: https://themagazine.synpulse.com/en/2019_09_19_genomics-study_gene-therapy/

The growing popularity of gene therapy earned much attention from all over the
world such that, other possible applications of this technique is being explored through
researches just like enhancement of height, intelligence, athletic ability, address infertility
and also its use in the field of cosmetology.
Although gene therapy has promising results, we always asked the question, “Do the
potential benefits of gene therapy outweigh the potential risks?

Please click/ visit the link below:

Watch this video clip: https://www.youtube.com/watch
You tube AGTC: Gene
?v=xOQFJJOBGM0
Therapy Explained

READ

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LEARNING ACTIVITY 1. Diseases and Gene Therapy

(Lesson Check – Up)

List down the diseases/disorders that can be treated by Gene Therapy using the table
below. Submit your output in word or PDF format at ______________________.

Types of Disease/Disorder Diseases/Disorders

1. Monogenic Disorders

2. Polygenic Disorders

3. Infectious Diseases

For more information related to the activity, browse the link:

http://www.genetherapynet.com/JoomlaTest2/index.php?option=com_content&view=article
&id=164:diseases-treated-with-gene-therapy-&catid=97:patient-information&Itemid=14

LESSON 2. Bioethics of Gene Therapy

At present, the type of therapy being undertaken is somatic gene therapy where the
DNA is inserted to a somatic cell and is not passed on to the offspring. Germline therapy on
the other hand, the DNA is inserted into a reproductive cell in which it can be passed on to
the offspring. The controversy lies in the latter type since the affected individuals are the
unborn ones who will be experiencing the unexpected consequences of the technique. The
following are some ethical questions surrounding gene therapy (Genetics Home Reference,
2017).

 How can “good” and “bad” uses of gene therapy be distinguished?

 Who decides which traits are normal and which constitute a disability or disorder?
 Will the high costs of gene therapy make it available only to the wealthy?
 Could the widespread use of gene therapy make society less accepting of people
who are different?
 Should people be allowed to use gene therapy to enhance basic human traits such
as height, intelligence, or athletic ability?

Many controversial issues arise on gene therapy and the government of United
States does not fund research studies on human germline gene therapy.

Please click/ visit the link below:

To know more about the
Pros and Cons of Gene www. biolyse .ca›gene-
Therapy therapy –pros and cons
READ

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LEARNING ACTIVITY 2. Let’s See Your Insights!

(Lesson Check – Up)

Position Paper. Make a clear stand if you would allow yourself for gene
therapy without its 100% assurance of effectiveness or future negative side effect. Write
your output in word or pdf format and be sure to submit it on __________________ at
_______________or email it at _______________________________________________.

Rubrics for Position Paper

Exemplary (5) Proficient (4) Emerging (3) Developing (2)
Criteria
Strong Reasons Position is Position is supported Position is Reasons are
supported with well with a minimum of supported with a weak and/or
developed and three distinct reasons. minimum of three repetitive. They
thought out reasons Reasons are distinct reasons. need to be
(minimum of three). developed, but more Reasons are developed and
Reasons are well analysis and developed, but are explained further.
developed, but conclusions are more general and
analysis and needed. need to be
conclusions need to developed further.
be strengthened.
Depth and Position is Position is supported Position needs to Paper lacks valid
Variety of supported with a with a variety of be supported with and accurate
Evidence variety of sources. sources. Valid and more valid and information. Some
Adequate valid and accurate information accurate of the information
accurate information has been used information. Some is not relevant
has been used throughout the paper. of the information and does not
throughout the Most of the information may not be support the
paper. Information is is relevant and relevant and/or position.
relevant and supports the writer’s does not support
supports the writer’s ideas. A mix of general the thesis.
ideas. and specific
information is used.
Organization and Most ideas are well Organization of the Paper has some Paper has little
Mechanics organized and free paper needs to be organization but organization and
of mechanical strengthened. some diversions many diversions,
errors. Mechanical errors are or abrupt shifts in often shifting in
present throughout the purpose are purpose. Many
paper. present. Many mechanical errors
mechanical errors are present and
are present as transitions are not
well. Sentence utilized.
structure needs to
be strengthened.
Transitions Some transitional Too few transitions Too few Attempts made to
words and phrases used, or used transitions used. use appropriate
are used to connect correctly. transitions.
ideas and maintain
coherence between
paragraphs.

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 ASSESSMENT TASK

Case Study: Gene Therapy for Enhancement Purposes

Direction: Read carefully the presented case below and answer the questions that
follow. Write your output in word or pdf format and be sure to submit it on __________ at
_______________ or email it at _________________________________.

“Dr. Anderson specializes in a particular type of gene therapy that targets

Alzheimer’s Disease (AD). Neural degeneration and synapse loss in the brain are
characteristic of AD. Therefore, this gene therapy aims to protect neurons from
degeneration and enhance the function of any neurons that are remaining. Dr. Anderson
has two patients request her services. However, after an initial meeting with them, she is
unsure whether she should treat them both.

Alexis is a 50 year-old woman who has a family history of AD and is already

beginning to experience very mild symptoms of what she thinks is AD. She tells Dr.
Anderson that her mother was afflicted with AD. So, she knows first-hand the sadness and
frustration the family of an AD patient has to experience. Alexis has a husband and three
children and does not want to put them through the same difficult journey. Therefore, she is
requesting the gene therapy to reverse the small-scale symptoms she already has and
prevent the onset of the disease.

Kelly is a 21 year-old college student who is applying for medical school in the very
near future. Her academic history is strong but not exceptional. For this reason, Kelly fears
that she will not be accepted to the top medical schools. Kelly wants to attend medical
school so she can help underserved populations and work in impoverished areas that lack
good healthcare. She tells Dr. Anderson that she would like to receive the Alzheimer’s gene
therapy in hopes it will boost her memory and enhance neural function. Kelly believes a
good score on the MCAT will strengthen her application and enable her to fulfill her dream
of providing medical aid to the world’s neediest people.”

Questions:

1. What is Alzheimer’s Disease? What are the symptoms?

2. At what particular age will Alzheimer’s disease develop in an affected individual?
3. If you are Dr. Anderson, are you going to give the treatment to both Alexis and Kelly?
Why or Why not?
4. As a young student aiming to become a board topnotcher someday, would you
submit yourself for enhancement gene therapy to boost your memory to increase
your intelligence? Why or why not?

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Rubrics for Case Study

Developing
Exemplary (5) Proficient (4) Emerging (3)
Criteria (2)
Scope Best and most Important points Some Fail to make
(Quality of applicable points are presented important any important
Presented are presented while unnecessary points are points and
Content) while unnecessary content is left out. addressed, but analyze the
content is left out. not fully material
covered. presented in
the unit and
case study.
Originality Demonstrates Demonstrates Case study Case study
(Interpretation critical thinking critical thinking lacks some lacks critical
and about the topic about the topic and critical thinking thinking about
Application) and the student’s the student’s own about the topic the topic and
own impressions impressions and and the the student’s
and interpretations interpretations of student’s own own
of research. The research. The impressions impressions
research is not research is and and
merely presented, presented, could interpretations interpretations
but is interpreted be better of research. of research.
and applied to interpreted and The research The research
overall themes. applied to overall presented is not
themes. could be better interpreted and
interpreted and applied to
applied to overall
overall themes. themes.
Presentation Proper sentence Few or no errors Overlooked Overlooked
(Grammar structure, but sentence errors in several errors
and punctuation, and structure could be sentence in spelling,
Mechanics) spelling, no editing improved. structure, punctuation,
or revision punctuation, and/or
required and spelling. sentence
structure
showing
carelessness.
Integration Various themes Various themes 50% of the Themes and
(Synthesis) and concepts and concepts themes and concepts are
throughout the throughout the concepts not integrated
paper are paper are throughout the and
integrated and integrated and paper are incorporated.
incorporated to incorporated to integrated and Concepts are
form even stronger form strong incorporated. not presented
support. Concepts support. Concepts, 50% of as a whole.
are not presented overall, are concepts are
independently of presented as a presented as a
one another, but whole. whole.
as part of a whole.

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Gene therapy is designed to introduce genetic material into cells to

compensate for abnormal genes or to make a beneficial protein. It aims to
cure or treat genetic-related human illnesses. Somatic and germline gene
therapy are the types of gene therapy. Gene therapy techniques include
gene augmentation therapy, gene inhibition therapy, and gene therapy killing
of specific cells. With some ethical issues on gene therapy, there are some
pressing questions on whose authority or power to decide which human
traits could be altered and or other concerns are on the discriminatory effects
of those who may not or cannot avail gene therapy.

 REFERENCES

 Amarille, Anna Theresa M., et. al. 2018. Science, technology and Society. Malabon
City, Mutya Publishing House, Inc.
 Bioethics case study: Gene Therapy. Accessed 8 July 2020 at
https://www.asmscience.org/content/book/10.1128/9781555816100.chap40a
 Case Study on gene therapy for enhancement purposes. Accessed 8 July 2020 at
https://knowgenetics.org/case-study-gene-therapy-for-enhancement-purposes/
 Serafica, J. P. J., Pawilen, G. T., Caslib, B. N. B., & Alata, E. J. P,. Science,
Technology and Society, First Edition 2018, Rex Book Store, Inc. Manila
 What are the ethical issues surrounding gene therapy? Accessed 8 July 2020 at
https://ghr.nlm.nih.gov/primer/therapy/ethics

Science,Technology and Society (STS-GEC 6)

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