GENE THERAPY

SCIENCE, TECHNOLOGY & SOCIETY

PREPARED BY:
( GROUP 6 )
1990 - The first approved gene therapy case
at the National Institute of Health, UK. It was
performed on a four year old named Ashanti
DaSilva. It was a treatment for a genetic
defect that left her with an immune system
deficiency.
WHAT IS GENE THERAPY?

- A type of experimental treatment

in which foreign genetic material
( DNA or RNA) is inserted into a
person’s cells to prevent or fight
disease. Gene therapy is being
studied in the treatment of certain
types of cancer or diseases.
Basic Process of Gene Therapy
- Replacing a mutated gene that causes
Several
disease with a healthy copy of the gene
approaches to
gene therapy - Inactivating, or “knocking out,” a
are being mutated gene that is functioning
tested, improperly
including:
- Introducing a new gene into the body
to help fight a disease
 In general, a gene
cannot be directly
inserted into a
person’s cell. It must be
delivered to the cell
 1st is the
IDENTIFICATION
2nd is the
REPLICATION
3rd is the INSERTION
 TYPES
OF
GENE THERAPY
 Somatic Gene Therapy
- Obtaining blood cells with a
genetic disease then introduce a
normal gene into the defective cell
- Has to be done several times
because the effects does not last
long, can't pass to the next
 Germline Gene Therapy
- Involves genetic modification
of the gametes (reproductive cells,
the egg cells and the sperm cells)
that will pass to the next
generation
Vectors - vehicles that delivers the gene
to the cells

Viruses - have the natural ability to

deliver genetic materials into cells.

Retrovirus - inserts its genetic code

directly into the chromosomes of the host
cell
Adenovirus - introduce the
deoxyribonucleic acid (DNA) they carry to
the nucleus but not into the chromosomes

Fatty Liposomes and Directly injecting to

the skin cells or tumor cells - not as
effective as the viruses used.
 Ex Vivo - outside the living,
cells are modified outside the
body and then transplanted back
again.
In Vivo - inside the living,
vector can be injected or given
intravenously (IV) directly to
the specific tissue in the body
 Stem Cell Gene Theraphy
Stem Cell – are the mother cells that have
the potential to become any type of cell in
the body. They have the ability to self-
renew or multiply.
Ex vivo, isolating the stem cells and
genetically correcting them, then
transplanting it back in the body with the
 Embryonic Cells - cells that are self-
replicating, derived from human
embryos or human petal tissue and able
to differentiate into 3 primary germ cell
layers, and into any type of cell in the
Somatic Cells - bodyAny cell of the body
except sex cells. Can differentiate
only into different cell types present
in the tissue of their origin
 The Bioethics of Gene Theraphy
1. How can 'good' and 'bad' uses
of gene therapy be
extinguished.

2. Who decides which traits are

normal and which constitue a
3. Will the high costs of gene therapy
make it available only to 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
ADVANTAGES & DISADVANTAGES
List of the Advantages of Gene
Therapy
1. It offers the possibility of a
positive medical outcome.
2. 2. We can treat disease or
illness in meaningful ways.
3. 3. It could improve a
person’s life in other ways
4. We already know that it can be a
successful treatment option.

5. There are out-of-body treatment options

available.

6. Gene therapy could be useful in the

treatment of multiple diseases.
7. The medical impact of gene therapy can
create permanent results.

8. New treatment options are coming out all

of the time.

9. It can work in combination with cell

therapy techniques.
List of the Disadvantages of
Gene Therapy 1. There could be
unwanted immune system
reactions.
2. Current gene therapy methods
can sometimes target the wrong
cells.
3. The delivery viruses might
recover their ability to create
4. Gene therapies could cause a potential
tumor.

5. There is no guarantee that the gene

therapy will work .

6. Some patients might experience

incompatibility issues.
7. Humans could develop a resistance to
the gene therapy.

8. The cost of gene therapy is prohibitive to

a number of families.

9. Ethical concerns about gene therapy exist

10. It typically works best in single-gene
mutations.

11. There are funding challenges to

consider in this category.