GENE

THERAPY
 OBJECTIVES
⊷ Describe gene therapy and its
various form;
⊷ Discuss the prevalence of gene
therapy in daily life; and
⊷ Explore the opportunities that may
be opened by gene therapy in the
future.
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 What is
“ Gene
Therapy?
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Gene Therapy
Is one example of a scientific and
technological innovation that
made a breakthrough in the area
of human health. Before aspirin
was ever understood at the nano
level, it had been known for it’s
effects. Gene therapy is an
experimental technique that uses
genes to treat or prevent disease. 
Gene therapy can replace a gene
that causes a medical problem
with one that doesn’t, add genes
to help the body to fight or treat
disease, or turn off genes that are
causing problems.
How do Gene
Therapy work?
In order to insert new genes
directly into cells, scientists use
a vehicle called a “vector”
which is genetically engineered
to deliver the gene.
Certain viruses are often used
as vectors because they can
deliver the new gene by
infecting the cell.
The vector can be injected or
given intravenously (by IV)
directly into a specific tissue in
the body, where it is taken up
by individual cells.
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. Somatic cell therapy is
viewed as a more conservative,
safer approach because it affects
only the targeted cells in the
patient, and is not passed on to
future generations.
Somatic gene therapy can be
broadly split into two categories:
Ex vivo - cells are modified
outside the body and then
transplanted back in again
In vivo - genes are changed in
cells still in the bod.
Types of Gene
Therapy
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.
The appeal of germ line gene
therapy is its potential for
offering a permanent therapeutic
effect for all who inherit the
target gene.
Successful germ line therapies
introduce the possibility of
eliminating some diseases from a
particular family, and ultimately
from the population.
 Gene Therapy
ADVANTAGES DISADVANTAGES

⊷ Gene therapy could change the ⊷ Gene therapy does not have a reliable
perspectives that people have about delivery method.
disease. ⊷ Humans could develop a resistance to the
⊷ Gene therapy could be used in different gene therapy.
ways to improve life. ⊷ Gene therapy is a costly treatment option
⊷ Gene therapy allows us to treat the
“untreatable” diseases.
 Gene Therapy and
Stem Cell Treatment
GENE THERAPY STEM CELL TREATMENT

⊷ Gene Therapy can be defined as the use ⊷ Cell Therapy can be defined as the
of genetic material (usually infusion or transplantation of whole cells
deoxyribonucleic acid - DNA) to into a patient for the treatment of an
manipulate a patient's cells for the inherited or acquired disease.
treatment of an inherited or acquired
disease.
Gene Therapy
as science and
technology
Medicine is one example of
scientific and technological
innovation that made a
breakthrough in the area of
human health.
Gene therapy as one of the
great contribution of
technology to biology.
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 ⊷ Although gene therapy is a promising
treatment option for a number of diseases
(including inherited disorders, some types
of cancer, and certain viral infections), the
technique remains risky and is still under
study to make sure that it will be safe and
effective.

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 Is Gene
Therapy
safe?
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 ⊷ Gene therapy is under study to determine whether it
could be used to treat disease. Current research is
evaluating the safety of gene therapy; future studies will
test whether it is an effective treatment option.
⊷ Several studies have already shown that this approach
can have very serious health risks, such as toxicity,
inflammation, and cancer.
⊷ Because the technique are relatively new, some risks
may be unpredictable; however, medical researchers,
institutions, and regulatory agencies are working to
ensure that gene therapy research is as safe as possible.
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 Gene Therapy
 Gene therapy is a treatment or cure for disorders
caused by mutated genes.
 It involves adding a normally functioning copy of
the gene(s) to enough affected cells to restore
normal function.
 Its goal is to help correct genetic disease by
providing functioning copy of gene.
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 The Process
of
Gene Therapy
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1 5
3

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 Types of Gene Therapy
Somantic Gene Germline Gene
Therapy Therapy
 Transfer of a section of DNA  Transfer of a section of
to any cell of the body that DNA to cells that produce
doesn’t produce sperm or eggs or sperms. Effects of
eggs. Effects of gene therapy gene therapy will be passed
will not be passed onto the onto the patient’s children
patient’s children. and subsequent
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generations.
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 Approaches in gene therapy
1. Ex Vivo Gene Therapy
 The target cells are removed from
the patient’s body, the genes are
fixed or replace and the cells are
put back into the person.

2. In Vivo Gene Therapy

 Direct delivery of genes into the
cells of particular tissue in the
20 body.
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 Gene Therapy Cures:

 Cystic Fibrosis
 Hemophilia
 Heart disease
 AIDS
 Cancer

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 WHAT WE SHOULD
KNOW ABOUT STEM
CELL TREATMENT IN
THE PH
Totel de Jesus

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 MANILA, Philippines- In recent years, it’s common to
hear beauty doctors in the Philippines offering stem
cells therapy as if it is a newfound medical fountain of
youth, the answer to all medical worries.

In various media platform, there are bombardment of

advertisements from various hospitals and clinics
packaging stem cell therapy as “cure-all” remedy.

With such medical breakthrough, it begs the question

on how equipped we are in offering such services. We
can’t help but ask the parameters on distinguishing the
real experts from seemingly fly-by-night clinics and
25 medical centers.
 Filipino-American doctor-layer Samuel Bernal, an
expert in the fields of regenerative medicine and
regulatory law, said the public should take a lot of
precautions.

“be careful. Number one, stem cell therapy is not a

cure-all (medical procedure),” he said in an interview
with the INQUIRER a few weeks after receiving the
2012 Presidential Award for Filipino Individuals and
Organizations Overseas at the Malacanan Palace.

“The goal of stem-cell therapy is not a longevity-anti-

aging program. Stem cell therapy is actually trying to
26 allow the body to heal itself,” he said.
 Educated at Johns Hopkins’ and Harvard Medical
School, Dr. Bernal is currently Professor Emeritus of
Medicine at the University of California-Los Angeles,
member of American Association of Clinical
Oncologists, the American Association of Cancer
Researchers, and a recognized cancer investigator with
the US National Institutes of Health.

As a lawyer, his legal expertise includes cases on

medical malpractice, medical product liability, as well
as Federal Drug Administration (FDA) and Federal
Trade Commission’s (FTC) regulations in the testing,
approval, advertising, and marketing of health
products.
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 He explained tem cell treatment requires “analyzing at
the molecular level what is causing he problem or
(what could be done) to prevent the problem.”

“stem cells are just a small part of the molecular

approach to medicine.”

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 THANK
YOU!
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