THE ASPECT OF
GENE THERAPY
LEARNING
OBJECTIVES:
At the end of this lesson, the students should
be able to;
• Describe gene therapy and its various
forms: and
• Assess the issues potential benefits and
detriments to global health.
• 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 Hospital
stated that they were able to restore basic hearing
in genetically deaf mice using gene therapy. The
Boston Children 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 "Anc 80" which
• 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 patients
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
THE BASIC
PROCESS
G C
• There are several approaches to gene therapy.
These are the following (Fliester, 2017)
• ·Replacement of mutated gene that causes disease
with a healthy copy of 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
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.
·Somatic Gene Therapy
·Germ-Line Gene Therapy
SOMATIC GENE
THERAPY
Involves the manipulation
of genes in cells that will
be helpful but not
inherited to the next
generation (Nimsergern,
1988).
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 ca 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 stem cells and somatic stem cells.
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 implanted into
woman (Crosta,2013).
SOMATIC STEM CELLS
Are cells that exist throughout the body after embryonic
development and are found inside of different types of tissue.
Tissues such as the brain, bone marrow, blood, blood vessels,
skeletal muscles, skin, and the liver.
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, germ-
line 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 illness. There are two forms
of gene therapy. One si 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 the
questions on whose authority
or power to decide which
human traits should be altered;
other concern are on the
discriminatory effects of those
who may not or cannot avail
gene therapy.
THANK YOU!
Quiz
Direction: answer the following questions in ½ sheet of pad paper.
1. Is a potential method to either treat or cure genetic-related human
illnesses.
2. When was the human gene therapy was actually first realized
3-5. give the several approaches to gene therapy.
6. It involves the manipulation of genes in cells that will be helpful but
not inherited to the next generation?
7. Which involves the genetic modification of germ cells or the origin
cells that will pass the change on to the next generation.
8. Are mother cells that have the potential to become any types of
cell in the body.
9. Are derived from a four-day-old human embryo that is in
blastocyst phase of development.
10. are cells that exist throughout the body after embryonic
development and are found inside of different types of tissues.
