G MODULE 10

e Gene Therapy Vs Stem Cell Therapy

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e Learning Outcomes
AtTthe end of this lesson, the learner should be able to:

h•• Define what a gene is

Differentiate gene therapy from stem cell therapy.

e•• Explain the importance of gene therapy in the future.

Demonstrate processes of gene therapy.

r • Discuss examples of genetic diseases.

INTRODUCTION
a
Genetic risks to health come from a variety of sources and predicting those risks can be much
p complicated than simply assessing disease inheritance patterns. The majority of health
more
conditions are not related to a single genetic cause but result from a combination of inherited and
y
environmental influences. Our topic will highlight risk factors and provide risk assessment tools,
which can improve healthcare decision making.
v chromosomes and genes provide instructions for our bodies, any changes to these
Because
structures have the potential to cause genetic disease and negatively impact health. We will
s
discuss the types of genetic diseases, how these diseases are inherited, and the potential heath
outcomes.
S
Understanding Gene Therapy

Thet expression of genetic conditions is not always as simple as the amount of DNA
(deoxyribonucleic acid) present, or the inheritance patterns of a single gene disorder. There are
e factors that determine how one’s genotype (genetic makeup) correlates to one’s
many
phenotype(physical manifestations). Among these are penetrance, expressivity, mosaicism, and
m
X-inactivation.

C
Penetrance is a measure of how often a disease genotype correlates to the disease phenotype.
For example, in a completely penetrant disease, every individual who is genetically positive for the
e
disease would have clinical manifestations of said disease. In contrast, for diseases with
incomplete penetrance, some individuals who are genetically positive for that disease would have
clinical manifestations of said disease. In contrast, for diseases with incomplete penetrance, some
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individuals who are genetically positive for that disease may be entirely asymptomatic.

l
Organisms inherit genetic information in a variety of ways that result in continuity of structure and
function between parents and offspring. Suppose you as a child have a genetic disease-one that
T
could be cured by diets or a similarly straightforward therapy. Wouldn’t the parents want the
disease diagnosed? Or take another example, perhaps two people are getting married. Wouldn’t
if hbeneficial to know what chance their offspring have of inheriting one or several harmful genetic
traits? We are now armed with the different advance procedures like the PCR, Southern blotting,
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RFLP mapping, and DNA Sequencing. Different laboratories have already developed the ability
to provide a very detailed look at our genetic materials. Is there a necessity to apply all these

r
techniques to all of us? What harm might it do to have a complete picture of our genes? In totality,
these procedures do not insert new genetic material into cells, no changes of any kind are made.
We are only provided with information about what is already there.

Cases of prenatal or perinatal diagnosis of a birth defect or an enzyme deficiency is one situation
where DNA diagnosis could be of immediate beneficial use. What must be emphasized is that the
information to be garnered by the new technology should be private and used by choice by the
individual whose DNA has been read. A person's DNA sequence to my mind should be protected
by law from distribution to other parties without the individual's specific permission. This goes the
same with physicians who have obtained such information, who should also treat it as they do any
privileged material.

The introduction of foreign genes into organisms doesn't seem to be dangerous. The resulting
organisms don't appear to be better able to reproduce than any others. Attempts to remedy human
disease by somatic gene therapy also don't appear to pose ethical dilemmas. But the introduction
of genes into the human germ line does. The diagnostic procedures that genetic engineering
allows are very powerful. Before anything else, it will be relatively easy to correlate the presence
of various DNA polymorphisms with disease and other hereditary characteristics.

GENETIC ENGINEERING
Gene therapy involves altering the genes inside your body’s cells in an effort to treat or stop the
disease.

It involves the removal of a desirables gene from a cell, adding it to the gene structure of a bacterial
cell and replacing the recombinant DNA into the bacterial cell. The bacteria then have the
capability to produce the protein produces by the original animal or plant cell.

GENE THERAPY
Knowledge of genetics is making possible new fields of health care. An example is the finding genes
that may have mutations that can cause disease will aid in the development of preventive measures
to fight disease. Substances such as hormones and enzymes, from genetically engineered.

organisms may reduce the cost and the side effects of replacing missing body chemicals.
The gene therapy has become commonly used term in biomedical research. This process involves
the splicing of functional genes into cells that contains defective, nonfunctional genes for a
particular trait. The clinical techniques used to accomplish gene therapy are still being worked out.
However, they involve research into methods of introducing the functional genes into the person
or organism affected by defective gene.
Simply, gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or
improve your body’s ability to fight disease.

DISEASED PEOPLE CURED BY FIXING THEIR GENES

Putting genes into plants and domestic laboratory animals is one thing. What about using genetic
engineering to modify humans, in particular to cure human disease? There are two quite different
procedures that are being debated under this heading, and they need to be discussed separately.
The first procedure is to insert foreign genetic material into the germ line-into sperm or eggs-where
they could be passed on to succeeding generations. The second procedure is to use genetic
engineering to insert foreign genes into somatic cells-into any cells except sperm or eggs and their
progenitors-in an effort to correct some genetic defect.
Some questions may arise like: Does the process work? Will it yield a better quality of life for the
treated individual? Were the patients, parents or guardians given informed consent? Treating
human genetic disease by purposely changing the genes that are passed on to succeeding
generations seems potentially dangerous and ethically unsound. It is dangerous precisely
because of the problems brought up at the very beginning. There is a great possibility of some
great interaction between the introduced gene and the remainder of the genome. The
consequences of this ignorance fall onto succeeding generations-on innocent children or fetuses.
On the other hand, if the genetic engineer and the physician make a mistake in somatic therapy,
the person who suffers is the patient, who should have been appraised of dangers before the
therapy was begun.

MODULE 10
ASSESSMENT TASK
• Define genes.
• Differentiate gene therapy from stem cell therapy.
• What is stem cell? Why it is important in medicine?
Discuss the uses.
• Explain the importance of gene therapy in the future.
• How does Genetic Engineering work?
• What are the Ethical issues of Genetic Engineering?