Gene and Stem Cell Therapy

Week 13-14 - Bioengineering and Biotechnology 2

Second Semester, SY 2023-2024
Intended Learning At the end of this lessons, students should be able

Outcomes to:

1 DIFFERENT FORMS Describe gene therapy and stem cell and its forms.

Understand the different approaches and types of both stem

2 TYPES & APPROACHES
cells therapy and gene therapy.

Assess the issues potential benefits and detriments to global

3 PROS & CONS
health.

4 CURRENT To discover recent studies and breakthrough on gene therapy and

BREAKTHROUGHS stem cells.

5 MORAL & FUTURE

Discuss the moral and ethical implications, identify the effects,
ISSUES particularly in the context of science, health and economy of
gene- & stem cell therapy and potential future impacts on
society, health and economy.
 CONTENTS

1 GENE THERAPY: DEFINITION &

HISTORY 4 STEM CELL THERAPY: DEFINITION,
TYPES, & USES

2 GENE THERAPY: TYPES AND

APPROACHES 5 STEM CELL THERAPY: IMPORTANCE,
DONATING & HARVESTING

3 GENE THERAPY: STATUS & ISSUES

6 STEM CELL THERAPY: ISSUES & CONCENS
 4
Definition, Types & Uses

STEM
❑ What are Stem Cells
❑ Characteristics and
Properties
❑ Types of Stem Cells

CELLS
❑ Stem Cell Therapy
❑ Uses of Stem Cells

THERAPY
WHAT ARE STEM CELLS?
These inner cell mass from a blastocyst. These stem cells
can become any tissue in the body. for example: neuron,
chondrocyte, enterocytes, red blood cells, muscle, fat or
epithelial cells

Stem cells are cells from which all other cells with
specialized functions are generated. Under the right
conditions in the body or a laboratory, stem cells divide
to form more cells called daughter cells.

At the point when a stem cell isolates, every daughter

cell can possibly either remain an stem cell or turn into
another sort of cell with specific capacity, for example,
muscle cell, red platelet or cerebrum cell.
 MORE ABOUT STEM CELLS
They are essential cells that replace damaged cells or cells lost due to diseases
They are the earliest cells of the cell lineage in all tissues and are found in both embryonic and adult organisms
They provide a continuous supply of new cells that make up the tissues and organs of animals and plants.
They have been of great interest as a therapeutic method for various diseases and conditions.
Stem cells in the embryonic organism are present in the inner cell mass of the blastocyte, which then differentiates
into all other cells in the body.
Stem cells in adults, however, are localized to specific areas within the body (ex., in the bone marrow and the
gonads)

The ability to self The ability to

regenerate differentiate
THE TWO DEFINING
CHARACTERISTICS
STEM CELL CHARACTERISTICS/PROPERTIES

Unspecialized or undifferentiated‘ (‘blank cells’)

All stem cells are present as a mass of cells that differentiate later
during their period of division.
Proliferation and renewal
Stem cells, of all origins, are capable of dividing and renewing
themselves for long periods of time.
These cells undergo a period of cell proliferation while preserving the
undifferentiated state.
Differentiation
Have the potential to give rise to specialized cells that together make
up different tissue types.
These cells can either be PLURIPOTENT or MULTIPOTENT
TYPES OF STEM CELLS
Totipotency cell: Ability to differentiate into cell lineages from
all three germ layers: mesoderm, endoderm, and ectoderm
including placental cell.
Ex. cells from early (1-3 days) embryos

Pluripotent cell: Ability to differentiate into cells from all three

germ layers with the exception of placenta.
Ex. some cells of blastocyst (5 to 14 days)

Multipotent cell: Ability to differentiate into a limited number

of types from one germ layer.
Ex. Fetal and adult stem cells, umbilical cord stem cells,
hematopoietic stem cells, and mesenchymal stem cells.

Unipotent cell: Ability to produce cells of their own type.

Ex. in the testes spermatogonia give rise only to sperm
WHAT IS STEM CELL THERAPY

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.
USES OF STEM CELLS

Tissue regeneration
Tissue regeneration is probably the most important use of stem cells. Until
now, a person who needed a new kidney, for example, had to wait for a donor
and then undergo a transplant. There is a shortage of donor organs but, by
instructing stem cells to differentiate in a certain way, scientists could use them
to grow a specific tissue type or organ.

Cardiovascular disease treatment

In 2013, a team of researchers from Massachusetts General Hospital reported
in PNAS Early Edition that they had created blood vessels in laboratory mice,
using human stem cells.
Within 2 weeks of implanting the stem cells, networks of blood-perfused
vessels had formed. The quality of these new blood vessels was as good as
the nearby natural ones.
The authors hoped that this type of technique could eventually help to treat
people with cardiovascular and vascular diseases.
USES OF STEM CELLS

Brain disease treatment

Doctors may one day be able to use replacement cells and tissues to treat
brain diseases, such as Parkinson’s and Alzheimer’s.
Researchers have already tried differentiating embryonic stem cells into these
types of cells, so treatments are promising.

Blood disease treatments

Doctors now routinely use adult hematopoietic stem cells to treat diseases,
such as leukemia, sickle cell anemia, and other immunodeficiency problems.
Hematopoietic stem cells occur in blood and bone marrow and can produce all
blood cell types, including red blood cells that carry oxygen and white blood
cells that fight disease.
 4
Importance, Donating & Harvesting

STEM
❑ Importance of this therapy
❑ Planarians
❑ Where do Stem Cells
naturally come from?

CELLS
❑ Donating or Harvesting
Stem Cells

THERAPY
IMPORTANCE OF STEM CELLS

Stem cells can replace diseased or damaged cells

1

Stem cells allow us to study development and genetics

2

Stem cells can be used to test different substances (drugs and chemicals)
3
PLANARIANS USED IN STEM CELLS STUDIES
Planarians are flatworms that are capable of
profound regenerative feats dependent upon a
population of self-renewing adult stem cells called
neoblasts.
Regeneration in planarians offers a paradigm for
understanding the molecular and cellular control of
the repair and regeneration of animal tissues, and
could provide valuable insights for the safe use of
stem cells to repair damaged, diseased and ageing
human tissues with little or no regenerative
capacities.
The goal of stem cell research to know the
fundamental stem cell concepts of regeneration
using Planaria as a model organism.
WHERE DO STEM CELLS NATURALLY COME FROM?

Stem cells originate from two main sources: adult

body tissues (Adult Stem Cells) and embryos
(Embryonic Stem Cells). Scientists are also working
on ways to develop stem cells from other cells, using
genetic “reprogramming” techniques.
Adult stem cells are undifferentiated cells, found all
through the body after improvement, that increase by
cell division to renew dying cells and recover harmed
tissues.
Embryo Stem Cells are cells derived from the inner
cell mass of the blastocyst prior to implantation. They
are pluripotent and have an unlimited capacity for
self-renewal and the ability to differentiate into any
somatic cell type.
DONATING OR HARVESTING STEM CELLS

A. Frozen Embryos: To be used for

stem cell research, are thawed and
cells from the pluripotent inner cell
mass is removed and grown in a Petri
dish in a research laboratory. Under
the right conditions, these embryonic
stem cells can self-renew (make more
of themselves by dividing) indefinitely.
DONATING OR HARVESTING STEM CELLS

B. Therapeutic Cloning: is a
technique used to create stem cells
that are a genetic match to a donor.
It has been used to clone non-human
animals, the most famous being the
sheep, Dolly. Therapeutic
cloning has been negatively
associated with the idea of human
reproductive cloning, but the majority
of scientists do not support the
reproductive uses of this procedure in
humans.
DONATING OR HARVESTING STEM CELLS

Bone marrow: These cells are taken under a general

anesthetic, usually from the hip or pelvic bone. Technicians
then isolate the stem cells from the bone marrow for
storage or donation.

Peripheral stem cells: A person receives several

injections that cause their bone marrow to release stem
cells into the blood. Next, blood is removed from the body,
a machine separates out the stem cells, and doctors return
the blood to the body.

Umbilical cord blood: Stem cells can be harvested from

the umbilical cord after delivery, with no harm to the baby.
Some people donate the cord blood, and others store it.
This harvesting of stem cells can be expensive, but the
advantages for future needs include:
1. The stem cells are easily accessible
2. Less chance of transplanted tissue being rejected if it
comes from the recipient’s own body.
DONATING OR HARVESTING STEM CELLS

Adult Cells (Skin Cells): can be

reprogrammed to behave like
undifferentiated human embryonic
stem cells, producing induced
pluripotent stem cells, or iPS cells.
Pluripotent cells are able to develop
into any of the hundreds of cell types
in the human body, such as muscle,
nerves, cartilage, blood or bone.
 4
Issues & Controversy

STEM
❑ Issues Concerning Stem
Cells
❑ Controversy

CELLS
THERAPY
ISSUES CONCERNING STEM CELLS

Ethical and Social Issues. It's a topic that brings up some big questions such as:
• When does human life begin?
• Can we have the benefits of stem cell research without using embryos?
• Might stem cell research make human cloning more likely and does this matter?
• Is it a good idea to do stem cell research at all when there are people suffering
from preventable diseases (like TB) in developing countries?
• We need to decide what we think and feel about embryos.
• Is an embryo just a ball of cells?
• Does it have the potential to be human but is not yet human?
• Or is an embryo a human being right from fertilization?

In the UK, stem cell research is regulated by the Human Fertilization and Embryology
Authority (HFEA). They allow limited research to do with fertility treatment relating to
IVF (In Vitro Fertilization), and research into 'serious diseases'
CONTROVERSY ON STEM CELLS

There has been some controversy about stem cell research. This mainly relates to
work on embryonic stem cells.

Use of embryos for stem cells

The argument against using embryonic stem cells is that it destroys a human
blastocyst, and the fertilized egg cannot develop into a person. Nowadays, researchers
are looking for ways to create or use stem cells that do not involve embryos.

Mixing humans and animals

Stem cell research often involves inserting human cells into animals, such as mice or
rats. Some people argue that this could create an organism that is part human.
In some countries, it is illegal to produce embryonic stem cell lines. In the United
States, scientists can create or work with embryonic stem cell lines, but it is illegal to
use federal funds to research stem cell lines that were created after August 2001.
CONTROVERSY ON STEM CELLS

Stem cell therapy and FDA regulation

Some people are already offering “stem-cells therapies” for a range of
purposes, such as anti-aging treatments. However, most of these
uses do not have approval from the U.S. Food and Drug
Administration (FDA)Trusted Source. Some of them may be illegal,
and some can be dangerous. Anyone who is considering stem-cell
treatment should check with the provider or with the FDA that the
product has approval, and that it was made in a way that meets with
FDA standards for safety and effectiveness.
 CONTROVERSY ON STEM CELLS

Would you agree

on their opinions?
If not what will be
your stand?
REFERENCES

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6724388/pdf/jbra-23-03-0197.pdf
https://media-exp1.licdn.com/dms/document/C4E1FAQEBDNi_terjBA/feedshare-document-
pdf-
analyzed/0/1618896996327?e=2147483647&v=beta&t=Wl4IKLCtw1GvaWvlBECtxLvmlflxjJM-
_bcGIBoRUmY
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7987258/pdf/btt-15-67.pdf
https://www.medicalnewstoday.com/articles/323343#uses
https://www.nwabr.org/sites/default/files/STEM_CELL_CURRICULUM_1109.pdf
https://www.pnas.org/doi/pdf/10.1073/pnas.1306562110
https://stemcellres.biomedcentral.com/track/pdf/10.1186/scrt474.pdf
https://www.medicalnewstoday.com/articles/323343#controversy
https://medlineplus.gov/genetics/understanding/basics/gene/#:~:text=A%20gene%20is%20th
e%20basic,more%20than%202%20million%20bases.
THANK YOU