Lesson 11

LESSON 11
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 hopes 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 of Gene Therapy
There are several approaches to gene therapy. These are the
following (Fliesler,2017):
1. Replacement of mutated gene that causes disease with a healthy copy
of the gene.
2. Inactivation of a mutated gene that is functioning improperly.
3. 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
(Nimsergern,1988). The other form of gene therapy is called germline 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 the following according to 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 research on human germline gene
therapy.

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LESSON 11

Two Types of Gene Therapy

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