GENE THERAPY

SCIENCE, TECHNOLOGY & SOCIETY

PREPARED BY:
( GROUP 4 )
WHAT IS GENE THERAPY?

is an experimental technique
that uses genes to treat or
prevent disease. In the future, this
technique may allow doctors to
treat a disorder by inserting a gene
into a patient's cells instead of
using drugs or surgery.
 ALSO, it is when DNA is introduced
into a patient to treat a genetic
disease. The new DNA usually
contains a functioning gene to correct
the effects of a disease-causing
mutation.
 Who created gene therapy?
The first approved gene
therapy clinical research in
the US took place on 14
September 1990, at the
National Institutes of Health
(NIH), under the direction of
William French Anderson
 How is DNA transfer done?

A carrier called a vector must be used

to deliver the therapeutic gene to the
patient's target cells. The vector acts
as a vehicle to carry the new DNA into
the cells of a patient with a genetic
disease.
 TYPES
OF
GENE THERAPY
 Somatic gene therapy
transfer of a section of DNA to
any cell of the body that doesn’t
produce sperm or eggs. Effects of
gene therapy will not be passed
onto the patient’s children.
 Germline Gene Therapy
transfer of a section of DNA to cells
that produce eggs or sperm. Effects
of gene therapy will be passed onto
the patient’s children and
subsequent generations.
ADVANTAGES & DISADVANTAGES
 Which diseases can be treated
with gene therapy?

Gene therapy holds promise

for treating a wide range
of diseases, such as cancer,
cystic fibrosis, heart disease,
diabetes, hemophilia and AIDS
Side effects of gene therapy

the patient's immune system

may react to the foreign vector,
causing fever, severe chills (called
rigors), drop in blood pressure,
nausea, vomiting, and headache.
These symptoms typically resolve
within 24-48 hours of the infusion.
•DNA mutations
•Immune response
•Viral spread
Reversion of the virus
to its original form
•Risk to offspring
•High cost
 Why Gene Therapy Is Important?

The goal of gene therapy is to cure or

slowdown a genetic disease by repairing
the damaged gene responsible for
the disease. To achieve this goal, gene
therapy requires the technology to insert
the missing “normal” gene into the DNA
of the patient's cells.