LESSON 3.

GENE THERAPY
A. Learning Outcomes: At the end of the lesson, you will be able to:
 describe gene therapy and its various forms;
 discuss the prevalence of gene therapy in daily life; and
 explore the opportunities that may be opened by gene therapy in the future.
B. Key Concepts
1. INTRODUCTION
When identifying the contribution of technology to biology, the first place to look would be the
field of human health. The whole vast concerns of human health including aging, disease treatment
and prevention, diet and general lifestyle have greatly benefited from technology.
Human gene therapy was first realized in 1971 when the first recombinant DNA experiments
were planned 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.
2. WHAT IS GENE THERAPY AND ITS PROCESS?
Gene therapy can be simply viewed as the insertion of foreign DNA into a patient's tissue that hope to
successfully eradicate the targeted disease
There are several processes of gene therapy. These are the following:
 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.
 A gene cannot be directly inserted into the human body it is with the use of a carrier or
vector. The vector which is normally used are viruses that have been genetically changed to
carry normal DNA.

Figure 3. Process of gene therapy.

3. APPLICATION OF GENE THERAPY
 It is used in the replacement of genes that cause medical ill-health
 The method generally destroys the problem causing genes
 It helps the body to fight against diseases by adding genes to the human body
 This method is employed to treat diseases such as cancer, ADA deficiency, cystic fibrosis, etc.
4. TYPES OF GENE THERAPY
 SOMATIC GENE THERAPY involves the manipulation of the genes in cells that will be helpful
to the patient but not inherited to the next generation.
 GERM-LINE GENE THERAPY involves the genetic modification of germ cells or the ongin
cells that will pass the change on to the next generation.
 STEM CELL GENE THERAPY are mother cells that have the potential to become any type of
cell in the body. The characteristic of stem cells is its ability to self-renew or multiply while
maintaining the potential to develop into other types of cells.
 Figure 4: Stem Cell
5. SOURCES OF STEM CELLS
 EMBRYONIC STEM CELL- derived from a four or five-day old human embryo.
 SOMATIC STEM CELL- these are cells that exist throughout the body after embryonic
development and are found inside of different types of tissue.
6. THE BIOETHICS OF GENE THERAPY
There are ethical issues involved in gene therapy. Some of the inquires cited are (Genetic Home
Reference, 2017):
 How can -good and bad uses of gene therapy be distinguished?
 Who decides which traits are normal and which constitute a disability or disorder?
 Will the high costs of gene therapy make it available only to the wealthy?
 Could the widespread use of gene therapy make society less accepting of people who are
different?
 Should people be allowed to use gene therapy to enhance basic human traits such as height
intelligence, or athletic ability?