Gene and
Stem Cell
Therapy
Prepared by Group 3
Today, we
will...
Discuss the Gene and Stem Cell
Therapy
Discuss its effects to the society
Present events or global issues that
affects these two therapies
Ethical issues
What is gene
defined as a treatment strategy that utilizes
genetically manipulated stem cells to transfer
desired genes into a patient's body, aimed at
and cell addressing genetic disorders and multifactorial
conditions such as neoplasia and infectious
therapy? diseases..
• These therapies are important because they offer
long-term or even permanent solutions to Gene therapy involves altering
diseases rather than just relieving symptoms. or replacing faulty genes with
• The development of gene and stem cell therapy healthy ones to correct genetic
began in the late 20th century. disorders
• Gene and stem cell therapies are being Stem cell therapy, on the other
developed and applied in advanced research hand, uses special cells that
centers and hospitals across the world. Countries can develop into many
such as the United States, Japan, South Korea, different cell types to repair or
replace damaged tissues or
and the United Kingdom are leading in
organs.
biotechnology research.
• Gene therapy works by inserting a healthy copy
Both therapies aim to restore
of a defective gene into a patient’s cells using a normal body function and cure
carrier called a vector (often a harmless virus). diseases that were once
Once inside, the new gene produces the correct considered untreatable..
protein needed by the body.
• Stem cell therapy involves harvesting stem cells
(from bone marrow, umbilical cord, or embryos),
growing them in the lab, and injecting them into
Gene and
Stem Cell
Therapy
Video
Presentation
Effects of Gene and Stem Cell
Therapy on society
Effects on Humans
Positive Effects:
• Treatment of Genetic Diseases - These
therapies can cure or reduce diseases like
cancer, diabetes, heart disease, and genetic
disorders.
• Improved Quality of Life - People who receive
successful treatments can live longer, healthier,
and more active lives.
• Advancement in Medicine - It leads to new
discoveries in healthcare and encourages
innovation in medical science.
Negative Effects:
• High Cost - These treatments are very
expensive and not accessible to everyone.
• Possible Side Effects - There’s still a risk of
unintended changes in DNA that might cause
Effects on Environment
Positive Effects:
• Healthier Populations - Reducing diseases in
humans and animals can lead to more stable
ecosystems and less medical waste.
• Biotechnology Advancement - Gene and
stem cell research can help grow organs,
improve crops, or heal ecosystems through
science.
Negative Effects:
• Uncontrolled Genetic Changes - If
genetically modified organisms (GMOs) or
altered genes escape into nature, they might
disrupt ecosystems.
• Loss of Biodiversity - Overuse of genetic
engineering could lead to less natural diversity
in plants and animals.
• Ethical Environmental Issues - Some people
worry about unnatural manipulation of life
forms, which could harm the balance of nature.
Updates Gene Therapy
Updates
about
The FDA and EMA have
approved new gene therapies for
sickle cell disease and beta
Gene and thalassemia, including the first
CRISPR-based therapy called
Stem Cell
Casgevy.
Scientists can now edit genes
Therapy directly inside the body, making
treatment easier. However, the
FDA is investigating two deaths
linked to a gene therapy for
Duchenne muscular dystrophy.
Researchers are testing gene
therapies for heart disease,
blindness, and brain disorders like
Huntington's disease.
Updates Stem Cell Therapy
Updates
about
There are over 100 clinical
trials using stem cells to treat
diseases, mainly focusing on eye,
Gene and brain, and cancer treatments.
Stem Cell
China has approved its first
stem cell therapy product, which
uses stem cells from umbilical
Therapy cords.
While stem cell therapy
seems safe so far, doctors are still
monitoring patients for long-term
effects.
Scientists are combining
gene editing with stem cell therapy
to make treatments more precise
and safer
Present events/global issue
that affects the gene and
stem cell
High cost and therapy
limited access
Many gene therapies (and advanced cell therapies) are extremely
expensive, which means a lot of people around the world can’t
benefit even if the therapy exists. For example:
Some gene therapies cost millions of US dollars for a single
patient.
Most of the research, clinical trials and approved therapies are in
high-income countries; low- and middle-income countries face big
barriers in infrastructure, cost, regulatory capacity.
Even for patients within richer countries, logistical issues like
travel to specialist centers, social support, and network access
create obstacles.
Present events/global issue
that affects the gene and
stem cell
Unproven therapy
therapies and regulatory gaps
In the stem-cell / regenerative medicine space, there’s a big
challenge with clinics offering treatments that haven’t been fully
proven safe or effective, and in many places regulatory oversight
is weak or being skirted.
Many so-called stem cell “treatments” are offered without rigorous
clinical trials or regulatory approval.
These treatments are sometimes marketed directly to patients,
especially those with serious diseases and few options, which
raises risks of physical harm, wasted money, disappointment.
Regulatory bodies in some countries are only now stepping up
enforcement or clarifying rules (for example a US court ruling
strengthened the ability of the Food and Drug Administration to
Ethical Issues
Stem cell therapy issues
• Moral status of the embryo - Research using
embryonic stem cells is controversial because it
typically requires the destruction of a human
embryo. This raises questions about when life
begins and the moral status of an embryo.
• Risk of over-promising - Patients with severe
illnesses are often targeted by unproven and
unregulated stem cell clinics, leading to
significant financial and health risks due to the
promotion of therapies that have not been
scientifically validated.
• Potential for cloning - The use of induced
pluripotent stem cells (iPSCs) raises ethical
concerns about their potential use in human
cloning and creating human-animal chimeras.
Ethical Issues
Gene therapy issues
• Germline editing - Altering the DNA of germline
cells (sperm, eggs, or embryos) would result in
permanent, heritable changes that could be
passed down to future generations. This raises
significant ethical questions about eugenics,
unforeseen side effects, and the potential to
create a society that is less accepting of human
variation.
• Somatic vs. germline therapy - The distinction is
crucial, as somatic gene therapy affects only
the individual patient and cannot be passed on,
while germline therapy has implications for all
future descendants.
Ethical Issues
Gene therapy issues
• Enhancement versus treatment - There is a
concern about using gene editing for non-
therapeutic enhancements, such as increasing
intelligence or athletic ability, rather than for
treating disease. This could lead to a form of
"neo-eugenics" and exacerbate social
inequalities.
• Safety and predictability - Risks include off-
target mutations that could cause new health
problems, and the potential for irreversible
changes once a therapy is administered, as
there is often no "off-switch".
Thank you
for
listening!