The Aspects of Gene Therapy Lecture Notes

THE ASPECTS OF GENE THERAPY
LESSON OVERVIEW
This lesson will discuss Gene Therapy concepts and its potential benefits and
disadvantages to global health.
DESIRED LEARNING OUTCOMES

At the end of the lesson, the students should be able to:
1. Describe gene therapy and its various forms
2. Assess the issues of potential benefits and detriments to global health
Defective genes were related to many human diseases. These diseases cannot be
cured by traditional methods like taking readily available medicines. Gene therapy is a
potential method to either treat or cure genetic – related human illnesses. In 2015, a team of
researchers at the Harvard Medical School and the Boston Children’s hospital has restored
the hearing of genetically deaf mice thru gene therapy. The research team in Boston Children’s
Hospital also reported that they bring back a higher level of hearing - down to 25 decibels
which is the same as a whisper. The “Anc80”, is an improved gene therapy vector developed
at the Massachusetts Eye and Ear which enables the transfer of genes to the inaccessible
outer hair cells when introduced into the cochlea.
In 1971, human gene therapy was first realized. That year, the first recombinant DNA
experiments were planned. This is done by simply inserting foreign DNA into a patient’s tissue
that hopes to remove the targeted disease. It was inspired by the success of recombinant
DNA technology which occurred over the last 20 years. Without a doubt, gene therapy is the
most promising yet possible unfavorable medical field being studies.
THE BASIC PROCESS

The following are several approaches to gene therapy:
• Replacement of mutated gene that causes disease with a healthy copy of the genes
• Inactivation of a mutated gene that is functioning improperly
• Introducing a new gene into the body to help fight a disease
In general, a gene cannot be directly inserted into a human gene or cell. A gene is
usually inserted into another gene using a carrier or vector. Nowadays, viruses are used,
after it has been genetically changed, to carry normal human DNA. It has been identified
that viruses have a way of encapsulating the genes in cells that will be helpful to the patient
but not inherited to the next generation.
TWO TYPES OF GENE THERAPY

The idea of gene therapy is based on correcting a disease at its root; fixing the
abnormal genes that appear to lead to certain diseases
1. Somatic Gene Therapy
This therapy involves the manipulation of genes in cells that will help the patient but
not inherited to the next generation
2. Germ-line Therapy
This therapy involves the genetic modification of germ cells or the origin cells that
will pass the change on to the next generation
STEM CELL GENE THERAPY

Stem cells are mother cells that have the potential to become any type of cell in the
body. They have the ability to self–renew or multiply while maintaining the potential to develop
into other types of cells. They can become cells of the blood, heart, bones, skin, muscles,
brain, among others. A stem cell has different sources but all types of stem cells have the
same capacity to develop into multiple types of cells.
Stem cells are derived from different sources. Two of which are embryonic and somatic
stem cells.
1. Embryonic Stem Cells

A stem cell-derived from a four to a five-day-old human embryo that is in the blastocyst
phase of development. The embryos are usually extras that have been created in vitro
fertilization (IVF) clinics where several eggs are fertilized in a test tube then implanted
into a woman.
2. Somatic Stem Cells
A stem cell that exists throughout the body after embryonic development and found
inside of different types of tissue can be found in tissues in the brain, bone
marrow, blood, blood vessels, skeletal muscles, skin, and the liver.
Until a stem cell is activated by disease or tissue injury, it will remain non-dividing for
years. These stem cells can divide or self – renew indefinitely, enabling them to generate a
range of cell types from the originating organ or even regenerate the entire original organ. It
is generally thought that adult or somatic stem cells are limited in their ability to
differentiate based on their tissue of origin, but there is some evidence to suggest that they
can differentiate to become other cell types.
THE BIOETHICS OF GENE THERAPY

Ethical issues that concern gene therapy are:
1. How can good and bad uses of gene therapy be distinguished?
2. Who decides which traits are normal and which constitute a disability or disorder?
3. Will the high costs of gene therapy make it available only to the wealthy?
4. Could the widespread use of gene therapy make society less accepting of people
who are different?
5. Should people be allowed to use gene therapy to enhance basic human traits such
as height, intelligence, or athletic ability?
The first death associated with gene therapy occurred on September 18, 1999, at the
University of Pennsylvania. Jesse Gelsinger was participating in a clinical trial, a biomedical
experiment for evaluation of safety and efficiency of a therapy for a disease. Gelsinger, who
was 18 years old at the time of the treatment, had a deficiency of ornithine transcarboamylase,
an important enzyme in the metabolism of ammonia. Patients with this rare metabolic disorder
must maintain a low-protein diet and take a series of medicines to avoid ammonia poisoning
in the blood stream. The gene therapy Gelsinger took triggered a chain reaction in his immune
system, resulting in hepatic and respiratory failure, and consequently, his death four days after
being treated.
Another controversy involves germline therapy. As discussed, germline

therapy is the genetic modification of germ cells that will pass the change on to the next
generation. A lot of questions on the effects of gene alteration on the unborn child and the next
generation, since the alteration can be passed on.
Effects on the Environment

Although the positive impacts of gene therapy could be enormous, there are many
questions raised that needs to be answered. New organisms created by genetic engineering
could present an ecological problem. One cannot predict the changes that a genetically
engineered species would make on the environment. The release of a new genetically
engineered species would also have the possibility of causing an imbalance in the ecology of
a region just exotic species would do. An accident or an unknown result could cause several
problems. An accident in engineering the genetics of a virus or bacteria for example could
result in a stronger type, which could cause a serious epidemic when released. This could be
fatal in human genetic engineering creating problems ranging from minor medical problems,
to death.
Effects on Human
Looking at the fact that genetic engineering employs viral vector that carries functional
gene inside the human body; the repercussion are still unknown. There are no clues as to
where functional genes are being placed. They may even replace the important genes, instead
of mutated genes. Thus, this may lead to another health condition or disease to human. Also,
as defective genes are replaced with functional gene, then it is expected that there will be a
reduction in genetic diversity and if human beings will have identical genomes, the population
as a whole will be susceptible to virus or any form of diseases.
Antibiotic Resistance
Genetic engineering often uses genes for antibiotic resistance as "selectable markers."
Early in the engineering process, these markers help identify cells that have taken up foreign
genes. Although they have no further use, the genes continue to be expressed in plant tissues.
Most genetically engineered plant foods carry fully functioning antibiotic-resistance genes.
The presence of antibiotic-resistance genes in foods could have lethal effects.
Therefore, eating these foods could reduce the effectiveness of antibiotics to fight disease
when these antibiotics are taken with meals. More so, the resistance genes could be
transferred to human or animal pathogens, making them impervious to antibiotics. If transfer
were to occur, it could aggravate the already serious health problem of antibiotic resistant
disease organisms.
Ethical and Social Issues

"Playing God" has become a strong argument against genetic engineering. Several
issues have also been raised as regards the acceptance of this technology. These concerns
range from ethical issues to lack of knowledge on the effects genetic engineering may have.
One major concern is that once an altered gene is placed in an organism, the process cannot
be reversed. The production of medicines through the use of genetically altered organisms
might develop extremely infectious forms that could cause worldwide epidemics.

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THE ASPECTS OF GENE THERAPY

DESIRED LEARNING OUTCOMES

THE BASIC PROCESS

TWO TYPES OF GENE THERAPY

STEM CELL GENE THERAPY

1. Embryonic Stem Cells

THE BIOETHICS OF GENE THERAPY

Another controversy involves germline therapy. As discussed, germline

Effects on the Environment

Ethical and Social Issues

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