Lesson 2
TITLE: The Aspects of Gene Therapy
INTENDED LEARNING OUTCOMES
After studying this module, you are expected to:
Describe gene therapy and its various forms; and
Enumerate the types of diseases that was eradicated by Medical
Science.
Differentiate the two forms of gene therapy
INTRODUCTION
Our genes are comprised of DNA, which are outlines to fabricate compounds and
proteins that make our body work. Apparently, people have somewhere in the range of 20,000
and 25,000 genes. We commonly get two duplicates of every gene from our folks. They
impact everything from the shade of our hair to our resistant framework; however qualities
aren't constantly constructed effectively. A little acclimation to them can change how our
proteins work, which at that point adjust the manner in which we inhale, walk or even
condensation food. Qualities can change as they experience acquired transformations, as
they age, or by being adjusted or harmed by synthetic compounds and radiation (acgt, 2020).
ACTIVITY
NAME IT:
Name at least 5 diseases that were considered as obsolete (non-existing) because of
medical breakthrough.
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ANALYSIS
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Despite the advancement of science, why is it that there are some diseases like cancer and
HIV, are still considered as incurable?
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ABSTRACTION
Introduction
• Gene therapy is an experimental technique that uses genes to treat or prevent disease.
In the future, this technique may allow doctors to treat a disorder by inserting a gene into
a patient's cells instead of using drugs or surgery Replacement of mutated gene that
causes disease with a healthy copy of the gene (Wikipedia, 2018)
• Gene therapy is a type of treatment designed to modify the expression of an individual’s
genes or to correct abnormal genes to treat a disease
Two Types of Gene Therapy
• Somatic gene therapy involves the manipulation of genes in cells that will be helpful
to the patient but not inherited to the next generation. (Serafica, Pawilin, et. al 2018)
• Germ-line gene therapy involves the genetic modification of germ cells or the origin
cells that will pass the change on to the next generation (Serafica, Pawilin, et. al 2018)
Stem Cell Gene Therapy
- Gene therapy is a novel therapeutic branch of modern medicine. Its emergence is a direct
consequence of the revolution heralded by the introduction of recombinant DNA
methodology in the 1970s.
- Gene therapy is still highly experimental, but has the potential to become an important
treatment regimen. In principle, it allows the transfer of genetic information into patient
tissues and organs. Consequently, diseased genes can be eliminated or their normal
functions rescued. Furthermore, the procedure allows the addition of new functions to
cells, such as the production of immune system mediator proteins that help to combat
cancer and other diseases (Thomas Swaka, 2017)
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The Bioethics of Gene Therapy
Because gene therapy involves making changes to the body’s set of basic instructions, it
raises many unique ethical concerns. The ethical questions surrounding gene therapy
include:
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?
Current gene therapy research has focused on treating individuals by targeting the therapy
to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed
to a person’s children. Gene therapy could be targeted to egg and sperm cells (germ cells),
however, which would allow the inserted gene to be passed to future generations. This
approach is known as germline gene therapy.
The idea of germline gene therapy is controversial. While it could spare future generations in
a family from having a particular genetic disorder, it might affect the development of a fetus
in unexpected ways or have long-term side effects that are not yet known. Because people
who would be affected by germline gene therapy are not yet born, they can’t choose whether
to have the treatment. Because of these ethical concerns, the U.S. Government does not
allow federal funds to be used for research on germline gene therapy in people. (McGill,
2020)
APPLICATION
DISCUSSION
You will be evaluated using the rubric below;
3
Description
Score Understanding Strategies Thinking Skills
Uses effective
Understanding of Uses logical thinking
4 strategies to get
concept is clearly evident to arrive at conclusion
accurate results
Uses appropriate
Understanding of the
strategies to arrive Shows thinking skills
3 concept is evident
at a result to arrive at conclusion
Has limited
Uses strategies Attempts to show
2 understanding of a
that are ineffective thinking skills
concept
Has a complete lack of Makes no attempt Shows no
1
understanding of concept to use a strategy understanding
What is gene therapy? What are the other various forms of gene therapy? Explain each.
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Should gene therapy be limited to medical concerns only or could it be used for aesthetic
purposes?
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Differentiate the two forms of gene therapy.
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LESSON SUMMARY
• Gene therapy gained a lot of commercial interest in the 1980s. In part this was because
many assumed such treatment would move swiftly and easily from proof of concept into
clinical trials. Such hopes, however, were dashed following the death of the first patient in
a gene therapy trial in 1999.
• It would take another decade before optimism about the therapy resurfaced. From 2008
onwards dozens of new start-ups began to be created around gene therapy.
• These were founded on the back of sponsorship from pharmaceutical companies and the
stock market. Just how much weight began to be attached to gene therapy can be seen
by the stock market’s valuation of Juno Therapeutics. In 2014, just one year after Juno
was set up, the company was valued at US$4 billion
