Learning Outcomes

The students will

 Discuss the different trends and issues in Biological
Science
 Assess the impact of the technologies and scientific
advancement on humanity.
 What is gene therapy?
Gene therapy is a medical
approach that treats or
prevents disease by correcting
the underlying genetic problem.
Gene therapy techniques allow
doctors to treat a disorder by
altering a person’s genetic
makeup instead of using drugs
or surgery.
 How does gene therapy work?
Gene therapy can compensate for
genetic alterations in a couple
different ways.

Gene transfer therapy introduces

new genetic material into cells. If an
altered gene causes a necessary
protein to be faulty or missing, gene
transfer therapy can introduce a
normal copy of the gene to recover
the function of the protein.
 How does gene therapy work?
Gene therapy can compensate for
genetic alterations in a couple
different ways.

Genome editing is a newer technique

that may potentially be used for
gene therapy. Instead of adding new
genetic material, genome editing
introduces gene-editing tools that
can change the existing DNA in the
cell.
Genetic material or gene-editing tools that are inserted directly into a cell
usually do not function. Instead, a carrier called a vector is genetically
engineered to carry and deliver the material. Certain viruses are used as
vectors because they can deliver the material by infecting the cell. The
viruses are modified so they can't cause disease when used in people.
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.
Alternately, a sample of the patient's cells can be removed and exposed to
the vector in a laboratory setting. The cells containing the vector are then
returned to the patient. If the treatment is successful, the new gene
delivered by the vector will make a functioning protein or the editing
molecules will correct a DNA error and restore protein function.
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)
 Why it's done?
Researchers are investigating several ways to do this, including:

 Replacing mutated genes. Some cells become diseased because certain genes work
incorrectly or no longer work at all. Replacing the defective genes may help treat
certain diseases.
 Fixing mutated genes. Mutated genes that cause disease could be turned off so that
they no longer promote disease, or healthy genes that help prevent disease could be
turned on so that they could inhibit the disease.
 Making diseased cells more evident to the immune system. In some cases, your immune
system doesn't attack diseased cells because it doesn't recognize them as intruders.
Doctors could use gene therapy to train your immune system to recognize the cells
that are a threat.
 RISKS
This technique presents the following risks:

 Unwanted immune system reaction. Your body's immune system may see the newly
introduced viruses as intruders and attack them. This may cause inflammation and, in
severe cases, organ failure.
 Targeting the wrong cells. Because viruses can affect more than one type of cells, it's
possible that the altered viruses may infect additional cells — not just the targeted
cells containing mutated genes. If this happens, healthy cells may be damaged, causing
other illness or diseases, such as cancer.
 Infection caused by the virus. It's possible that once introduced into the body, the
viruses may recover their original ability to cause disease.
 Possibility of causing a tumor. If the new genes get inserted in the wrong spot in your
DNA, there is a chance that the insertion might lead to tumor formation.
 What you can expect
in Gene Therapy?
 Currently, the only way for you to receive gene therapy is to participate in a clinical
trial. Clinical trials are research studies that help doctors determine whether a gene
therapy approach is safe for people. They also help doctors understand the effects of
gene therapy on the body.
 The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in
people have shown some success in treating certain diseases, such as:

 Severe combined immune deficiency  Hemophilia

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in
people have shown some success in treating certain diseases, such as:

 Blindness caused by retinitis pigmentosa  Leukemia

 What are stem cells?
 A stem cell is a cell
with the unique ability
to develop into
specialized cell types in
the body.
 Why is there such an interest
in stem cells?
Researchers hope stem cell studies can help to:
Increase understanding of how
diseases occur. By watching stem
cells mature into cells in bones, heart
muscle, nerves, and other organs and
tissue, researchers may better
understand how diseases and
conditions develop.
 Why is there such an interest
in stem cells?
Researchers hope stem cell studies can help to:
Generate healthy cells to replace
cells affected by disease
(regenerative medicine). Stem cells
can be guided into becoming specific
cells that can be used in people to
regenerate and repair tissues that
have been damaged or affected by
disease.
 Why is there such an interest
in stem cells?
Researchers hope stem cell studies can help to:
Test new drugs for safety and
effectiveness. Before using investigational
drugs in people, researchers can use some
types of stem cells to test the drugs for
safety and quality. This type of testing
will most likely first have a direct impact
on drug development for cardiac toxicity
testing.
 Where do stem cells come
from?
There are several sources of stem cells:
Embryonic stem cells. These stem
cells come from embryos that are 3
to 5 days old. At this stage, an
embryo is called a blastocyst and has
about 150 cells.
 Where do stem cells come
from?
There are several sources of stem cells:
Embryonic stem cells. These are
pluripotent (ploo-RIP-uh-tunt) stem
cells, meaning they can divide into
more stem cells or can become any
type of cell in the body. This
versatility allows embryonic stem
cells to be used to regenerate or
repair diseased tissue and organs.
 Where do stem cells come
from?
There are several sources of stem cells:
Adult stem cells. These stem cells are
found in small numbers in most adult
tissues, such as bone marrow or fat.
Compared with embryonic stem cells, adult
stem cells have a more limited ability to
give rise to various cells of the body.
 Where do stem cells come
from?
There are several sources of stem cells:
Adult cells altered to have properties
of embryonic stem cells. Scientists
have successfully transformed
regular adult cells into stem cells
using genetic reprogramming. By
altering the genes in the adult cells,
researchers can reprogram the cells
to act similarly to embryonic stem
cells.
 Where do stem cells come
from?
There are several sources of stem cells:
Perinatal stem cells. Researchers
have discovered stem cells in amniotic
fluid as well as umbilical cord blood.
These stem cells have the ability to
change into specialized cells.
Why is there a controversy about
using embryonic stem cells?
 Embryonic stem cells are obtained from early-stage embryos — a group
of cells that forms when eggs are fertilized with sperm at an in vitro
fertilization clinic. Because human embryonic stem cells are extracted
from human embryos, several questions and issues have been raised
about the ethics of embryonic stem cell research.
 There are moral and ethical
issues related to using embryonic
stem cells. At present, the
Department of Health (DOH)
has decreed that the use of
embryonic stem cells in the
Philippines is illegal.
Why can't researchers use adult
stem cells instead?
 Although research into adult stem cells is promising, adult stem cells
may not be as versatile and durable as are embryonic stem cells. Adult
stem cells may not be able to be manipulated to produce all cell types,
which limits how adult stem cells can be used to treat diseases.
 What is stem cell therapy
(regenerative medicine) and how
does it work?
 Stem cell therapy, also known as regenerative medicine, promotes the
repair response of diseased, dysfunctional or injured tissue using stem
cells or their derivatives. It is the next chapter in organ
transplantation and uses cells instead of donor organs, which are
limited in supply.
 What is stem cell therapy
(regenerative medicine) and how
does it work?
 The specialized cells can then be implanted into a person. For example,
if the person has heart disease, the cells could be injected into the
heart muscle. The healthy transplanted heart muscle cells could then
contribute to repairing the injured heart muscle.
 Have stem cells already been
used to treat diseases?
 Yes. Doctors have performed stem cell transplants, also known as bone
marrow transplants. In stem cell transplants, stem cells replace cells
damaged by chemotherapy or disease or serve as a way for the donor's
immune system to fight some types of cancer and blood-related
diseases, such as leukemia, lymphoma, neuroblastoma and multiple
myeloma. These transplants use adult stem cells or umbilical cord blood.
 Knowing all of these, what is
your stand in Gene Therapy and
The use of Stem cells?
REFERENCES:

Website:
1. https://medlineplus.gov/genetics/understanding/therapy/genetherapy/
2. https://medlineplus.gov/genetics/understanding/therapy/procedures/
3. https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619
4. https://www.youtube.com/watch?v=evH0I7Coc54
5. https://www.youtube.com/watch?v=S-CvC4BAIIo
6. https://www.youtube.com/watch?v=kAtd9X29SdQ
7. https://www.youtube.com/watch?v=i_1kLfK1cP0
8. https://www.themedicalcity.com/news/what-are-stem-
cells#:~:text=There%20are%20likewise%20moral%20and,found%20in%20tissues%20already%20differentiated.