Chapter 3 Lesson 5 The Aspects of Gene Therapy1

Chapter 3 Lesson 5 THE ASPECTS OF GENE THERAPY
Medical science has detected many human diseases related to defective genes. These
types of diseases are not curable 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 stated that they were able to restore basic hearing in genetically deaf mice using
gene therapy. The Boston Children's Hospital research team also reported that they have
restored a higher level of hearing-down to 25 decibels which is actually equivalent to a
whisper. They used an improved gene therapy vector developed at the Massachusetts Eye
and Ear that was identified as "Anc80" which enables the transfer of genes to the inaccessible
outer hair cells when introduced into the cochlea (Fliesler, 2017)
Human gene therapy was actually first realized in 1971 when the first recombinant DNA
experiments were planned. It can be simply viewed as insertion foreign DNA into a patient's
tissue that hope to successfully eradicate the targeted disease. It was actually 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 possibly unfavorable medical field being
studied.
The Basic Process
There are several approaches to gene therapy. These are the following (Fliesler, 2017):
 Replacement of mutated gene that causes disease with a healthy copy of the gene
 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 inserted
into another gene using a carrier or vector. At present, the most common type of vectors 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
(Science Daily, 2017).
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.
There are essentially two forms of gene therapy. One of which is called somatic 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 (Nimsergen, 1988).
The other form of gene therapy is called germ-line gene therapy which involves the genetic
modification of germ cells or the origin cells that will pass the change on to the next generation
(Your Genome, 2017).
Stem Cell Gene Therapy
Stem cells are mother cells that have the potential to become any type of cell in the body.
One of the main characteristics of stem cells is their ability to self-renew or multiply while
maintaining the potential to develop into other types of cells. Stem cells can become cells of
the blood, heart, bones, skin, muscles, brain, among others. There are different sources of
stem cells 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.
The embryonic stem cells are derived from a four- or five-day-old human embryo that is in
the blastocyst phase of development. The embryos are usually extras that have been created
in IVF (in vitro fertilization)clinics where several eggs are fertilized in a test tube then implanted
into a woman (Crosta, 2013).
The somatic stem cells are cells that exist throughout the body after embryonic
development and are found inside of different types of tissue. These stem cells have been
found in tissues such as the brain, bone marrow, blood, blood vessels, skeletal muscles, skin,
and the liver. They remain in a non-dividing state for years until activated by disease or tissue
injury. 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 (Crosta, 2013).
The Bioethics of Gene Therapy
There are ethical issues involved in gene therapy. Some of the inquiries cited are (Genetics
Home Reference, 2017):
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?
Another controversy involves the germline therapy. As discussed, germline therapy is

genetic modification of germ cells that will pass the change on to the next generation. There
are a lot of questions on the effects of the gene alteration to the unborn child and the next
generation, since the alteration can be passed on. In the United States, the government does
not fund researches on human germline gene therapy.
SUMMARY
Gene therapy is a method that may treat or cure genetic-related human illnesses. There
are two forms of gene therapy. One is somatic gene therapy which involves the manipulation
of genes in cells that will be helpful to the patient but not inherited to the next generation. The
other is germline gene therapy which involves the genetic modification of germ cells or the
origin cells that will pass the change to the next generation.
There are many ethical issues on gene therapy. Some of these issues are about questions
on whose authority or power to decide which human traits should be altered; other concerns
are on the discriminatory effects of those who may not or cannot avail gene therapy

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Chapter 3 Lesson 5 The Aspects of Gene Therapy1

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Chapter 3 Lesson 5 THE ASPECTS OF GENE THERAPY

The Basic Process

Two Types of Gene Therapy

Stem Cell Gene Therapy

The Bioethics of Gene Therapy

1. How can “good" and “bad” uses of gene therapy be distinguished?

Another controversy involves the germline therapy. As discussed, germline therapy is

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