*** SEMI-FINALS ***

LESSON 3: GENE THERAPY

Intended Learning Outcomes:

At the end of the lesson, the student should be able to:

 describe gene therapy and its various forms

 discuss the benefits and risks of gene therapy

 analyze the ethical issues surrounding gene therapy

INTRODUCTION

In the field of medicine, medical researchers are continuously looking for different ways
of treating genetic disorders and gene-related illnesses. With the advent of molecular biology,
the causes of genetic disorders were now being traced and defective genes are now detected. A
deeper understanding of the molecular components of the body such as genes, their functions,
locations and control mechanisms paved way for coming up of a clearer technique in treating
disorders. These ushered the development of treating genetic disorders called gene therapy.
The initial aim of gene therapy was the correction of inherited genetic diseases.

GENE THERAPY

Gene therapy is when a DNA is introduced into a patient to treat a genetic disease. The
new DNA contains a functioning gene to correct the effects of a disease causing mutation.
It is also an experimental technique that uses genes to treat or prevent disease. This technique
may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using
drugs or surgery.

TYPES OF GENE THERAPY

1. Somatic gene therapy – transfer of a section of DNA to any cell of the body that doesn’t
produce sperm or eggs.
2. Germline gene therapy – transfer of a section of DNA to cells that produces sperm or
eggs.

GENE THERAPY TECHNIQUES

There are several ways by which gene therapy is administered. This includes the following:

1. Gene augmentation therapy. It is designed for diseases caused by loss of function of a

gene, introducing extra copies of a normal gene may increase the amount of normal gene
product to a level where the normal phenotype is restored.
2. Gene inhibition therapy. This is suitable for the treatment of infectious diseases, cancer
and inherited diseases caused by the inappropriate gene activity. The aim is to introduce a gene
whose product either: inhibits the expression of another gene or interferes with the activity of
the product of another gene.

3. Gene therapy killing of specific cells. Genes are directed to the target cells and then
expressed so as to cause cell killing. Direct cell killing is possible if the inserted genes are
expressed to produce a lethal toxin (suicide gene) or a gene encoding a prodrug is inserted,
conferring susceptibility to killing by a subsequently administered drug.
Direct cell killing

Indirect cell killing

These are the current approaches being employed in gene therapy. The growing
popularity of gene therapy earned much attention from all over the world such that, other
possible applications of this technique is being explored through researches just like
enhancement of height, intelligence, athletic ability, address infertility and also its use in the
field of cosmetology.
BENEFITS AND RISKS OF GENE THERAPY

Gene therapy was first developed in 1972 and up to now it is still on the process of
refining the technique so as to ensure the success of its application. More sophisticated
instruments are being developed and more accurate processes are taken underway to reduce
the risks associated with it. For now, researchers and practitioners of gene therapy and the
whole of humanity are holding on to the following benefits this technique is giving to us.

1. Gene therapy is a source of hope.

2. The Effects of therapy are long-lasting and timeless.
3. Gene therapy can eradicate diseases and improve the quality of life.

Despite the potential benefits we get from gene therapy, there are still disadvantages
and risks associated with it considering that its target molecules are responsible for controlling
and regulating the functions of an organism. Some of these are:

1. There is no guarantee and it can go wrong.

2. There is a potential for developing resistance.
3. Gene therapy is a costly treatment option.

ETHICAL CONCERNS OF GENE THERAPY

At present, the type of therapy being undertaken is somatic gene therapy where the
DNA is inserted to a somatic cell and is not passed on to the offspring. Germline therapy on the
other hand, the DNA is inserted into a reproductive cell in which it can be passed on to the
offspring. The controversy lies in the latter type since the affected individuals are the unborn
ones who will be experiencing the unexpected consequences of the technique.

*** End of Lesson 3 ***