Gene Therapy

MODULE
GENE THERAPY 4
Learning outcomes:
At the end of this module the students must be able to:
a. describe gene therapy and its various forms; and
b. assess the issues potential benefits and detriments to global health;
Let’s try:
Check what you have by answering the following test without looking on the content or
browsing on the net. It will help you to determine what you have on your own understanding and
will guide you what are the thing that you need to know about the Gene therapy.
I. Multiple Choice Questions

Direction: Encircle the letter of the correct answer.
1. What is a somatic cell?
a. Body cell b. Sex cell c. Reproductive cell d. Nerve cell
2. When was recombinant DNA planned and the human gene therapy realized?
a. 1970 b. 1971 c. 1972 d. 1973
3. Does gene therapy can be directly insert in human gene?
a. Yes b. No c. Maybe d. Depend on the process
4. What type of gene therapy involves the manipulation of genes in cells that will be helpful to
the patient but not inherited to the next generation?
a. Somatic b. Germ-line c. Embryonic d. Stem
5. . What type of gene therapy involves the manipulation of genes in cells that will be helpful to
the patient and can be inherited to the next generation?
a. Somatic b. Germ-line c. Embryonic d. Stem
II. Word Bank

Direction: Define the following terms according to your own understanding.
1. Gene therapy
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2. Stem cell gene therapy
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MODULE
GENE THERAPY 4
Nature of Gene Therapy
Medical science has detected many human diseases

related to defective genes. These diseases are not
curable by traditional method like talking readily
medicines. Gene therapy is an experimental technique
that uses gene to treat or prevent disease. In the future,
this technique may allow doctors to treat a disorder by
inserting gene into a patient’s cell instead of using drugs
or surgery.
Human gene therapy was actually first realized in 1971

when the first recombinant DNA experiment were
planned. It can be simply viewed as insertion of foreign DNA into a patient’s tissue that hope to
successfully eradicate the targeted disease. It was inspired by the success of recombinant DNA
technology which occurred over the last 20 years.
The Basic Process of Gene Therapy

Researchers are testing several approaches to gene therapy which includes:
1. Replacing a mutated gene that causes disease with healthy copy of gene
2. Inactivating or knocking out a mutated gene that is functioning improperly.
3. Introducing a new gene into the body to help fight a disease.
In general, a gene therapy cannot be
directly inserted into a human gene or cell.
A gene is inserted into another gene using a
carrier or vector. At present, the most common
type of vector are viruses that have been
genetically changed to carry normal human DNA.
Viruses have evolved a way of encapsulating
and transporting their genes to human cells in
a pathogenic manner.
Two types of Gene Therapy
1. Germinal Gene Therapy – also called germ-line gene

therapy it involves the genetic modification of germ cells
(sperm or ova) that will pass the change on to the next
generation. During the production of gamete cells fuse to
form a zygote, which would divide and pass on the modified
gene into other cell of the body during the development of
offspring. In this way the therapy alters the genome of future
generations to come.
MODULE
GENE THERAPY 4
Although theoretically this could counteract hereditary disease, jurisdiction in
various countries such as Switzerland, Australia, and Germany prohibited use of
germline therapy due to fears of over unknown risks and long-term effects in the future
generations.
2. Somatic Gene Therapy – involves the manipulation of genes in cells that will be
helpful to the patient but not inherited to the next generation. Unlike germinal therapy,
somatic gene therapy only involves the insertion of therapeutic DNA into body cells and
not on the germ cells or gametes. This means any effects of the therapy are confined to
the individuals being treated and are not inherited by future generations.
The field of somatic gene therapy is surrounded by fewer ethical issues

compared with germline gene therapy although the therapeutic approach is also still in
the early stages of design and prone to obstacles. The first hurdle is successful
incorporation into the genome; integrating the modified gene into the wrong part of the
DNA could induced rather than prevent disease. Secondly, the desired gene needs to
be express. Thirdly, the gene expression needs regulating to prevent over expansion
triggering any disease.
Stem Cell Gene Therapy

Stem cell are mother cell that have the
potential to become any type of cell in the body.
One of the characteristics of stem cell is their
ability to self-renew or multiply while
maintaining the potential to develop into other
types of cells. Stem cell can become cells of
the blood, heart, bones, skin, muscle, brain,
among others. There are different sources of
stem cells but all type of stem cells have the same
capacity to develop into multiple type of cells.
Bioethics of Gene Therapy
There are ethical issues involved in gene therapy. Some of the inquiries cited are:
1. How can good and bad uses of gene therapy be distinguished?
2. Who decides which traits are normal and which constitute to disability or disorder.
3. Will the high cost of gene therapy make it available only to the wealthy?
4. Could the widespread use of gene therapy make society less accepting of peoples
who are different?
5. Should people be allowed to use gene therapy to enhanced basic human traits, such
as height, intelligence, or athletic ability?
MODULE
GENE THERAPY 4
Learning Activity time!
Acitivity 1. Make a flow chart of the basic process of gene therapy. Explain each part of the
process. You may use references and pictures. Use one short size bond paper.
Activity 2. At the space bellow make a Venn diagram and differentiate the two forms of gene
therapy.
Let’s check what you got!
1.Would you subject yourself for gene therapy without its 100% assurance and effectiveness or
future negative side effect?
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2. Should gene therapy be limited to medical concerns only or could it be used for aesthetic
purpose?
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MODULE
GENE THERAPY 4
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Gene Therapy

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MODULE

I. Multiple Choice Questions

II. Word Bank

Nature of Gene Therapy

Medical science has detected many human diseases

Human gene therapy was actually first realized in 1971

The Basic Process of Gene Therapy

Two types of Gene Therapy

1. Germinal Gene Therapy – also called germ-line gene

The field of somatic gene therapy is surrounded by fewer ethical issues

Stem Cell Gene Therapy

Bioethics of Gene Therapy

Let’s check what you got!

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