Gene Therapy: Science, Technology and Society

SCIENCE, TECHNOLOGY
AND SOCIETY
Gene
Therapy
Potential Benefits
EXPECTED OUTCOME
Students will:
1. Have a substantial knowledge of aspects
regarding gene therapy;
2. Know the importance of gene therapy,
Our Agenda especially in the fild of medicine; and
3. Appreciate the efforts and contributions of
for Today scientists to improve global health

standards.
What is
Gene Therapy?
AN EXPERIMENTAL TECHNIQUE
THAT USES GENES TO TREAT OR
PREVENT DISEASE
Genes
What is Gene - Small segments of deoxyribonucleic
acid (DNA), which are considered the
Therapy? biological units of heredity.
- Important in the determination of
an individual's traits.
- Flaws or abnormalities can lead to
disorders or diseases.
One of the emerging scientific

advances that provide new
treatment paradigm for genetic
disorders and diseases is the so-
called GENE THERAPY
Categorization of
Gene Therapy
DEPENDING ON TYPES OF CELL TREATED
Somatic Gene Therapy Germline Gene Therapy
Process of DNA transfer involves any cell This results in permanent changes that are
of the body that does not produce egg passed on to the patient's children and
cells or sperm cells. The effects of gene subsequent generations since this
therapy will not be passed on to the technique involves the cells of the
patient's offsprings. germline (eggs or sperm).
1. Gene Augmentation
Therapy
2. Gene Inhibition
Therapy
Approaches
to Gene 3. Killing of Specific
Therapy Cells
Two Sides of A
Coin:
ASSESSIG TEH POTENTIAL
BENEFITS AND DETRIMENTS OF
GENE THERAPY TO GLOBAL
HEALTH
Gene Therapy is making an attempt to
revolutionize the way diseases are treated.
Gene Therapy
Introduce a
Targets the actual
healthy gene via
source of the
the so-called
problem, the
vectors to replace
defected or muted
or repair such
gene.
defective gene.
Phase I Trial
"FIRST-IN-HUMANS" TRIAL
These studies focused on treating inherited

immune deficiencies, like severe combine
immunodeficiency (SCID) and adenosine
deaminase (ADA) deficiency, hereditary blindness,
hemophilia, beta-Thalassemia, as well as more
complex diseases, like cancer and cardiovascular
disorders.
September 14, 1990
National Institutes of Health Clinical Center
First Successful demonstration in trial of Gene Therapy
The technique was done to a 4-year old girl who had ADA deficiency.
To treat this, the doctors obtained White Blood cells from the
patient, allowed these cells to grow in the lab, and inserted them
with the working copies of the defective gene before returning
them into the patient's body.
The result was successful - the girl's immune system improved
40%. This, however, was temporary, since the procedure needs to
be performed again due to the geentically modified white blood
cells that are functional for a few months only.
Treatment of Hemophilia
Abnormal Bleeding and Poor Blood Clotting
Absence of certain clotting factors in the blood.
People with severe condition of hemophilia may loose large
amounts of blood from internal hemorrhage or even from a
minor cut.
The missing clotting factor, Factor IX, was successfully
transfered via viral vector to liver cells of patients who
participated in a small trial for gene therapy for this condition.
After treatment, most of the patients made at lest some Factor
IX, and they had fewer bleeding incidents.
In addition...
Gene Therapy has also been used to treat other
complex diseases.
Today, researchers are
engaged in many clinical
trials of the application of
Gene Therapy.
Despite the huge potential of Gene
Therapy to eradicate and prevent
heredetary diseases, as well as to
provide possible cure for AIDS,
cancer, and heart diseases, gene
Therapy is surrounded by issues
and controversies.
SAFETY
GENE EDITING ON HUMANS
Primary concern regarding
Gene Therapy
Drawbacks and
CHIMERIC ANTIGEN
RECEPTOR CELL THERAPY
Dangers of Gene HARMFUL MUTATIONS OF

Therapy THE DNA.
VIRUSES
GENETIC TESTING
REGULATION OF THE USES

OF GENE THERAPY
BOTTOMLINE:
Gene Therapy
This can bring out the changes to society,
positively or detrimentally. Its potential to
treat different diseases afflicting humans
could be limitless, but it could also give rise
to variety of problems, like safety concerns,
as well as ethical and moral issues.
Thank you!
GROUP 2 - CE1205
Reporters:
Estrada, Ashville G.
Castillo, Crizelle S.
Mentor:
Acorda, Tricia T.

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SCIENCE, TECHNOLOGY

for Today scientists to improve global health

One of the emerging scientific

Somatic Gene Therapy Germline Gene Therapy

These studies focused on treating inherited

Dangers of Gene HARMFUL MUTATIONS OF

REGULATION OF THE USES

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