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Module 12 - The Aspect of Gene Therapy

1) Gene therapy involves inserting healthy genes into patients' cells to treat diseases like cancer, diabetes, and genetic disorders. It has been used for decades in bone marrow transplants and is showing promise for other conditions. 2) The Philippines may establish ethical standards and promote investment in stem cell and gene therapies to become a medical tourism hub for cheaper treatments. 3) While gene therapy is being tested for currently incurable diseases, it remains risky and more research is needed to ensure safety and effectiveness. There are also ethical debates around who can access or modify genes and how genetic changes may impact future generations.

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Mark Daniel DeTorres Golisao
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231 views4 pages

Module 12 - The Aspect of Gene Therapy

1) Gene therapy involves inserting healthy genes into patients' cells to treat diseases like cancer, diabetes, and genetic disorders. It has been used for decades in bone marrow transplants and is showing promise for other conditions. 2) The Philippines may establish ethical standards and promote investment in stem cell and gene therapies to become a medical tourism hub for cheaper treatments. 3) While gene therapy is being tested for currently incurable diseases, it remains risky and more research is needed to ensure safety and effectiveness. There are also ethical debates around who can access or modify genes and how genetic changes may impact future generations.

Uploaded by

Mark Daniel DeTorres Golisao
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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MODULE 12

THE ASPECT OF GENE THERAPY

Lesson objective:
At the end of this module, the students should be able to:
1. Describe gene therapy and its various forms
2. Explore the opportunities that may be opened by gene therapy in the future.
3. Examine a bioethical issue from the viewpoint of various stakeholders.
4. Learn about the risks and potential outcomes involved in actual gene therapy
trials.

Gene therapy has been existence for almost half of century and is already the
standard care in certain procedures such as bone marrow transplants. The past decade
has seen this new and innovative science reach heights and has been proven to be
effective in treating diseases such as stroke, autism, Parkinson's, diabetes, spinal cord
injury and host of other ailments, both common and rare.
In 2013, La Union Rep. Eufranio Eriguel has introduced House Bill No. 212,
which would put up a “bioethics advisory board” that would establish “ethical standards”
governing the practice of stem cell therapy. Its purpose is to make the country a haven
for open technological innovation for stem cell, and will promote investment and
generate jobs. Further on, the Philippines may become known as hub for medical
tourism, with the surging increase of foreigners to come in for stem cell therapy that
some hospital in the country may offer at a much cheaper cost but with highest quality.

The Basic Process


Gene therapy is 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. Researchers are testing several
approaches to gene therapy, including:
 Replacing a mutated gene that causes disease with a healthy copy of the gene.
 Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
 Introducing a new gene into the body to help fight a disease.

Source: https://ghr.nlm.nih.gov/primer/therapy/procedures
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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.
Although gene therapy is a promising treatment option for a number of diseases, the
technique remains risky and is still under study to make sure that it will be safe and
effective. Gene therapy is currently being tested only for diseases that have no other
cures.

Types of Gene Therapy


There are two different types of gene therapy depending on which types of cells are
treated:

Source: https://socratic.org/questions/meiosis-occurs-in-
reproductive-cells-while-mitosis-occurs-in-somatic

A. Somatic gene therapy: transfer of a section of DNA to any cell of the body
that doesn’t produce sperm or eggs. Effects of gene therapy will not be
passed onto the patient’s children.

B. Germline gene therapy: transfer of a section of DNA to cells that produce


eggs or sperm. Effects of gene therapy will be passed onto the patient’s
children and subsequent generations.
C.
STEM CELL
Stem cells are special type of cells in the body that have the ability to differentiate
into other cell types. This ability allows them to replace cells that have died. Because of
this ability, they have been tapped to replace or control defective cells in patients

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who have certain diseases or defects. There are three sources of autologous adult stem
cells:
1. Bone marrow, harvested by aspiration, is used to treat patient undergoing
chemotherapy.
2. Blood, collected through leukapheresis - a process where blood is drawn from the
patient, passed through a machine that selects only the stem cells and   returns all
other components of the blood back to the patient. Bone marrow transplant and
blood stem transplantation is used to treat leukemia and lymphoma
3. Adipose tissue, have a capacity of self-renewal is harvested by liposuction. It is used
for clinical trials for the treatment of diabetes mellitus, liver disease, corneal lesions,
articular and cutaneous lesions, among others

Source: https://ghr.nlm.nih.gov/primer/therapy/procedures
a. Bone marrow stem cell b. blood stem cell

c. adipose stem cell


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

According to doctor-lawyer Samuel Bernal, an expert in the fields of regenerative


medicine and regulatory law, stem cell therapy is not a cure all medical procedure. Its
goal is not longevity-anti-aging program. Stem cell therapy is trying to allow the body to

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heal itself. The process needs an army of basic scientists in molecular biology,
biochemistry and biochemical engineering because every patient is unique. Model in
clinical trials in drugs doesn't apply to stem cell therapy. Gathering information from
other people may give you the clue but it doesn't give you the full information for each
patient.

The Bioethics of Gene Therapy


The ethical questions surrounding gene therapy includes (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 hereditary traits such
as height, intelligence, or athletic ability?
 Should people allowed to alter DNA of unborn babies
 Whose authority or power to decide which human traits should be altered on the
next generation
It is vital to understand the principles of bioethics and ethical science aligned with
their socio-ecological responsibility, so as to prioritize the health and welfare of man, in
order to properly utilize the natural resources and technology. Among the fundamental
principles of bioethics are respect for life in all its forms, and to ensure the maintenance
of life.
The law relating to the use of gene therapy is rarely debated in scientific circles,
because of great resistance on the use of these clinical trials, despite numerous
successes of stem cell. However, such perspectives are submerged in dubious
methods, and the effects of the projects now under study are not predictable. As stated
in the Universal Declaration of Human Rights; everyone has the right to life, but there is
the question of to what extent we can risk this right on behalf of still unfinished scientific
research.
As in other areas of research, validation of new therapeutic methods is closely
related to the development of clinical trials, and prior approval by local, national and
international ethics committees is, therefore, required. Some types of vectors, notably
adenoviral and retroviral vectors, have produced serious and even fatal side effects
and, therefore, security seems to be the main obstacle for the application of this type of
clinical intervention in hospitals and other public health care.
Not so far from the new technological possibilities applied to modern medicine, many
matters involving moral and ethics were raised with heated debate, especially on the
behavior of the professionals involved - including doctors, researchers, patients and
other people involved with the problems of medicine and public health.

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