Gene therapy

2. _
WHAT IS A GENE?
- Genes are regions of DNA that
code for proteins. Failure to
produce normal levels of functional
protein due to a defective gene
can result in disorders such as
haemophilia B, cystic fibrosis,
lysosomal storage disorders,
lipoprotein lipase deficiency
(LPLD) and some cancers.

4. Pathophysiological process such as in injury and wound
healing, chronic pain and arthritis, cancer and acquired
infectious such as human immunodeficiency virus type 1
(HIV-1) are now becoming realistic targets for this
promising therapeutic modality.
- The first authorized gene-transfer study took place at
the National Institutes of Health (NIH) in 1989. in this
maker study (see GENETICS MAKERS), tumor-infiltrating
lymphocytes were harvested, genetically tagged using a
retroviral vector and reinfused with the intention of
examining the tumor-homing capacity of

5. these cells . This landmark study provided the
first direct evidence that human cells could be
genetically modified and returned to a patient
without harm. Since then, over 2200 trials
have been approved, initiated or completed
worldwide, which are performed predominantly
in the United States. Most studies have
focused on cancers, with monogenic,
cardiovascular, and infectious diseases the next
most frequent indications.

6. MEDICINE
– is one example of a
scientific and technological
innovation that made a
breakthrough in the area
of human health.

7. GENE DELIVERY
TECH.
TARGET CELL
BIOLOGY
DISEASE
PATHOSHYSIOLOGY

9. 1. GENE DELIVERY TECHNOLOGY
- Gene delivery systems can be classified
into two broad categories: non viral
physicochemical approaches and
recombinant viral systems. The
comparative strengths of non viral
approaches include ease of chemical
characterization, simplicity and
reproducibility of production, larger
packaging capacity and reduced
biosafety risks.

10. 2. TARGET CELL BIOLOGY
- Each viral vector system possess a
unique set of biological properties, and
their use is governed largely by the
biology of the target cell. For example,
integration provides the molecular basis
for stable long-term gene expression as
would be required for the treatment of
genetic disease in replicating cell
populations.

11. 3. DISEASE PATHOPHYSIOLOGY
- Before a gene-therapy approach can be
considered feasible for a disease or physiological
process, a precise understanding of the underlying
molecular basis required. The requirement for
transient or persistent transgene expression must
also be considered. For example, the
extrachromosomal nature of adenoviral vectors
has the potential to limit the duration of gene
expression by delusional loss during cell division.
In some context, in which only transient gene
expression is required, such as anticancer gene
therapy, this may be positive attributed.

12. ISSUES, POTENTIAL BENEFITS AND
DETRIMENTS TO GLOBAL HEALTH
Over the last 20 years, the initial thoughts of gene therapy have been transformed into reality with
more than 175 clinical trials and 2,000 patients already treated. Yet with all the trials, there is still
no conclusive evidence for efficacy. Although the expectations have exceeded the initial success
of this relatively new field, important information has been gained from preclinical and clinical
trials. with this in mind, it is imperative to realized that with recent technological advances, gene
therapy for treating a wide variety of disease is likely to become a reality within the early part of
the next century. There are clearly a number of obstacles limiting successful gene therapy, but
the most difficult to overcome has been the inability to transfer the appropriate gene into a target,
non-germ-cell tissue, such that an appropriate amount of the gene product (usually a protein) is
produced to correct the disease. The cellyorganisms has develop powerful mechanisms to avoid
the accumulation of extraneous genetic material. The purpose of this session was to illustrate both
the highlights as well as hurdles that remain for developing successful clinical applications.

13. GROUP 1
Q: Some people say that the field of medicine is
business; which one is true? Field of medicine is
healing or selling? Why?
A: It actually goes both ways. One of the goal in the
field of medicine is to help sick people heal and
regain their well-being but nothing is for free,
because they will also buy some resources and raw
materials upon making medicines for people. So
basically, it is a give and take process.

14. GROUP 2
Q: IS GENE THERAPY A PROMISING TREATMENT?
A: Yes! In fact, there are already successful
treatments done this past few years. Example
of this is the successful gene therapy trial in
patient with x-linked chronic septic
granulomatosis last 2013.

15. GROUP 3
Q:IS IT POSSIBLE THAT THE GENE THERAPY
DELIVER BAD GENES INTO CELLS?
A: Gene therapy is done by experts and
professionals in the field of medicine.
However, gene therapy procedures are still on
the process of improvement so there could be
issues with cell division or replication that
limit the effectiveness of the treatment.

16. GROUP 4
Q: IN THE FUTURE, IS IT POSSIBLE FOR US TO COME UP
WITH A VACCINE FOR HIV?
A: There is currently no vaccine available that will prevent HIV
infection or treat those who have it. However, scientists are
working to develop one. Building on the findings on an earlier
study that found for the first time Albert Modestly, that a
vaccine could prevent HIV infection in 2016, an NIH-supported
clinical trial was launched to test a modified HIV vaccine. This
current vaccine trial, called HVTN-702, is testing whether an
experimental vaccine regimen safely prevent HIV infections
among south Africa adults. Hence, it is really possible for us to
come up with an effective and promising vaccine in the near
future.

17. GROUP 5
Q: DOES GENE THERAPY IS BEING APPLICABLE IN
HUMAN EXPIREMENT?
A: Gene therapy is an experiment itself! (hehehe :D)

18. GROUP 6
Q: WHAT DO YOU THINK IS/ARE THE DISADVANTAGE/S OF GENE
THERAPY?
A: Gene therapy is really a good way of treating people with illnesses.
However, most forms of gene therapy are still in the clinical research
stage. Gene therapy has several disadvantages such as follows:
1. Gene therapy does not have a reliable delivery method.
2. Gene therapy is an expensive procedure.
3. Gene therapy may not be able to adapt to a changing world.
4. Gene therapy will shift society towards new polarization.

19. GROUP 7
Q:HOW CAN PROTIENS BE USED BY GENE
THERAPEUTIC COMPANIES TO BETTER THE
DEVELOPMENT OF THEIR THERAPEUTIC PRODUCTS?
A: Proteins serves as an initial proof of principle of gene
based therapies. Therefore gene therapy can be considered
a form of protein therapy. However instead of the
therapeutic usage of the protein itself, gene therapy works
by placing into a cell a define gene to either replace a
defective gene or to increase the amount of a specific gene
in a targeted cell/tissue in order to produce a higher
amount of the desired protein.

20. GROUP 8
Q: HOW WILL THE THIRD WORLD COUNTRIES BENEFIT
IN THE GENE THERAPY WHEN THEY ARE SOMEHOW
LATE UPDATED WHEN IT COMES TO SCIENCE AND
TECHNOLOGY?
A: Honestly speaking, the third world countries can’t
benefit yet to any gene therapy procedures because they
are still economically unstable. Gene therapy is a very
expensive procedure. Even the “affordable” options in this
field start at to P200,000 per treatment. That’s why many
patient weight for clinical trials to begin, and then apply
for a spot in one to receive the help they need but most
health care insurance plans will not cover the cost of these
procedures because of their uncertainty.

21. GROUP 9
Q: FOR YOU, WHAT IS THE ESSENCE OG GENE
THERAPY TOWARDS ONE’S HEALTH?
“Gene therapy could change the perspectives that people
have about disease.”
A: Approximately 33M people are suffering from a disability
that is directly attributed to their genetic profile. The
promise of gene therapy is that it can reduce or eliminate
the pain and discomfort that these abnormalities cause.
80% of the diseases that we know impact human health in
negative ways have a genetic foundation. If we can replace
the cells or chromosomes that are at-fault, then it becomes
possible to offer relief.

22. GROUP 10
Q: IS THERE ANY RISK OF GENE THERAPY?
A: Retrovirus delivery systems are the most
common way for gene therapies to be delivered to
patients. The problem with this option is that the
enzyme used to encourage the transfer of genetic
data can be eliminated by the immune system
before it has the chance to work. There could be
issues with cell division or replication that limit
effectiveness of the treatment.

23. GROUP 12
Q:WHAT MAKES GENE REPLACEMENT THERAPY
DIFFERENT FROM OTHER GENE-BASED
THERAPY?
A: There is actually no other gene-based therapy
other than gene “replacement” therapy as of the
moment. But scientists are still undergoing several
clinical trials and experimental techniques to
improve ways of treating people in the field of
medicine.