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Lecture 09 - Biotechnology For Human Welfare-1
Lecture 09 - Biotechnology For Human Welfare-1
BIOTECHNOLOGY FOR
HUMAN WELFARE
A. STEM CELLS
What are stem cells?
They are the foundation cells for every organ,
tissue and cell in the body
Fetal SCs
Umbilical Cord SCs
Adult SCs
Sources of stem cells
1. Early embryo
newly fertilized egg produces a group of stem
cells called an embryo
these early stem cells are totipotent
Sources of stem cells
2. Blastocyst embryo
seven days after fertilization, embryo forms a
hollow ball-like structure called a blastocyst
embryonic stem cells in the blastocyst are
pluripotent
Sources of stem cells
3. Fetus
the embryo is referred to as a fetus after 8 weeks
of development
like embryonic stem cells, fetal stem cells are
pluripotent
Sources of stem cells
4. Umbilical cord
blood from the umbilical cord contains stem
cells genetically identical to the newborn child
umbilical cord stem cells are multipotent
Sources of stem cells
5. Adults
infants and children also have them
like umbilical cord stem cells, adult stem cells
are multipotent
Sources of stem cells
Research and clinical applications
of stem cells
Replace diseased specialized cells with healthy
cells, called cell therapy (e.g., Parkinson's disease)
Cell therapy – similar to organ transplantation,
except that the process consists of transplanting
cells instead of organs
Study of biological processes
development of the organism
progress of cancer
Drug discovery and development
Research and clinical applications of stem
cells
Stem Cell Therapy
Bone marrow (contains hematopoietic stem cells)
transplants
Any disease in which there is tissue degeneration
can be a potential candidate for stem cell therapies
Parkinson's disease
Alzheimer's disease
Spinal cord injury
Stroke
Burns
Heart disease
Type I diabetes
References
http://gslc.genetics.utah.edu/units/stemcells/
What is a Stem Cell? What are some different types of Stem
Cells?
http://www.lifesciences.umich.edu/research/featured/tutorial.
html
Animation from the University of Michigan. Very thorough.
Yu J and JA Thomson. 2006. Embryonic stem cells.
http://www.isscr.org/public/faq_printversion.html
http://www.csa.com/discoveryguides/stemcell/overview.php
B. Tissue
Engineering
Definition
Tissue Engineering is
the development and
manipulation of
laboratory-grown
molecules, cells, tissues
and organs to replace or
support the function of
defective or injured body
parts.
In practice the term has
come to represent
applications that repair or
replace structural tissues
(i.e., bone, cartilage, blood
vessels, bladder, etc.),
tissues that function by
virtue of their mechanical
properties.
http://cjmems.seas.ucla.edu
Final Direction
Transplantation science
Biomaterials
Biomedical engineering
References
Baboon
Pigs in xenotransplantation
Pigs are the animal of choice in xenotransplantation
research
inexpensive to raise
Pig heart
easy to breed
health compared to
baboons or monkeys
pigs are closest to Human heart
humans after apes and
monkeys
A very brief history
1682: The skull of an injured Russian
aristocrat was repaired with a bone
from a dog. Operation was a
success but angered the church.
1963-4: 12 patients received
chimpanzee kidney transplants in
New Orleans, US. Most failed
within two months but one
survived for nine months with no
sign of rejection.
1984: The Baby Fae case – A
newborn baby received a baboon
heart in Californina. Cyclosporine
(immunosuppressant) was used
and she lived for 20 days
1992: A four-drug cocktail assisted a
baboon liver transplant. The Baby Fae with hypoplastic left
patient died of a brain hemorrhage heart syndrome. Recipient of an
ABO-blood group mismatched
after 71 days. The type of rejection baboon heart
typical in cross-species
transplantation was not seen.
Issues/Concerns
Immune rejection
Microbiological barriers
Xenozoonoses
Issues/Concerns
1. Immune rejection
The transplanted organ is viewed as a foreign object
by the host's immune system, triggering a cascade of
immune reactions against the donor organ.
Recipient’s blood harbors antibody molecules that
bind to donated tissues
Attachment of these antibodies activates special
“complement” proteins in the blood, which triggers
the destruction of the graft.
Capillaries are destroyed leading to massive
hemorrhage.
Issues/Concerns
Immune rejection
Issues/Concerns
PPL Therapeutics
Ltd.
produced cloned pigs
(March 2000)
introduced a foreign
engineered disease-
free pigs that have
modified cell surface
molecules to reduce
problems of rejection.
Fostering tolerance / avoiding rejection
administration of
immunosuppressive
drugs (e.g.,
cyclosporine), but risks
of toxicity, infections
and other
complications still
remain
Modification of the
immune system with
bone marrow cells
from donor animal
genetic modification of
pigs
Issues/Concerns
2. Microbiological barriers
25 known diseases can be
contracted from pigs to
humans
Xenozoonoses
Infection of humans with
an animal pathogen
transmitted by
transplantation of animal
tissues or cells.
Pigs have PERVs, porcine
endogenous retroviruses
PERVS are similar to
HIV
shown to infect some
cells
tissues
organs
systems
Organization of the Human Body
cells
tissues
organs
systems
Cell types
Nerve cells
Blood cells
Cells of blood vessels
DNA - deoxyribonucleic
acid
gene – a specific sequence in the DNA that is
transcribed
Genetic diseases:
Type 1: Single locus (gene) is defective and
responsible for
the disease, 100% heritable.
examples: Sickle cell anemia
Hypercholesterolemia
Cystic fibrosis
Type 2: Polygenic traits, <100% heritable, may be
dependent on environmental factors and lifestyle.
examples: Heart disease
Cancer
Diabetes
Alcoholism
Schizophrenia
Criminal behavior
Definition of Gene Therapy
vivo
Protein expression
Cytosol
Endosome
Transcription
Lysosome (nucleases)
retroviruses
adenoviruses
Vectors in gene therapy
Viral vector
Non-viral DNA carriers:
Cationic liposomes:
- positively charged lipids interact with negatively
charged DNA
(lipid-DNA complex)
- traverse cell membranes
Advantages:
a. Stable complex
b. Can carry large-sized DNA
c. Can target to specific cells
d. Do not induce immunological reactions
Disadvantages:
a. Low transfection efficiency
b. Transient expression
c. Inhibited by serum
d. Some cell toxicity
E. Biopharmaceuticals
A Look at Recombinant Drugs
What are biopharmaceuticals?
Biopharmaceuticals are medical drugs produced
using biotechnology. They are proteins or
nucleic acids used for therapeutic or in vivo
diagnostic purposes.
Produced by means other than direct extraction
from a native (non-engineered) biological source.
Protein pharmaceuticals
The first “protein vaccine” was cow pox (Jenner,
1796). The first protein pharmaceutical was
insulin (Banting and Best, 1922).
http://www.aapa.org/clinissues/images/v http://www.tqnyc.org/NYC051907/
accinia_vaccine.jpg Insulin.jpg
Protein pharmaceuticals
Protein drug Animal Source
Insulin Pig or cattle
pancreas
Albumin Human blood
HGH (human growth Human brain
hormone)
Factor VIII Human blood
Calcitonin Salmon
Anti-venom Horse or goat
blood
Protein pharmaceuticals
Protein
pharmaceuticals from
blood
The body contains 6
L of blood
Blood is considered a
pharmaceutical
cornucopia
http://bloodbanker.com/images/Give-blood-
promotional-sign.jpg
Protein pharmaceuticals
Protein pharmaceuticals from blood
The body contains 6 L of blood
Blood is considered a pharmaceutical cornucopia
Examples include:
Factor VIII (for blood clotting-for hemophilia)
Factor IX (for blood clotting-for hemophilia)
Peptide drugs
Many hormones are actually small
peptides (2-40 amino acids) and as
such, peptides can be synthesized and
can be used as pharmaceuticals.
Protein pharmaceuticals
Peptide drugs
Examples:
Calcitonin, a thyroid hormone used to enhance
bone mass
Oxytocin, a pituitary hormone used to stimulate
labor
Vasopressin, also a pituitary hormone used as an
3110_102260_5.JPG
http://www.appdrugs.com/ProdJPGs/OxytocinLg.jpg
http://carnegieinstitution.org/first_light_case/horn/lesso
ns/images/bacteria.jpg
http://www.utoronto.ca/greenblattlab/images/a/yeast%20
1.jpg
http://www.bscb.org/softcell/images/mp_tripple.gif
http://www.molecularfarming.com/ediblevaccine.html
F. Bioremediation
biodiversity conservation
environmental monitoring
bioremediation
Bioremediation
Cleanup
involves “altering the availability
of nutrients, organisms, O2 and other
components in order to…
accelerate the natural process of
biodegradation”
Bioremediation
Biosorption
Microbial Degradation
Phytoremediation
Biosorption
- degradation of xenobiotic
compounds and petro chemicals by
bacteria or fungi
- microbes utilize these compounds as
source of energy and carbon
compounds for cell synthesis
Degradation of polychlorinated
biphenyls (PCBs)
Some bacteria that can degrade PCBs
- Flavobacterium
- Pseudomonas
- Arthrobacter
- Rhodococcus
- Clavibacter
Phytoremediation
SEQUESTRATION
phytoextraction
hyperaccumulation
phytovolatilization
phytostabilization
Hyperaccumulators
Biological weapon
infectious agents such as bacteria, virus, protozoa, or
fungi that are intentionally used to inflict harm to others
use of toxins or chemicals produced by microorganisms,
plants, or animals
invisible, microscopic, yet deadly
BIOTERRORISM: PAST, PRESENT,
AND FUTURE
4TH B.C. - Throw earthen pots with
serpents to enemies
6TH century B.C. –Poisons from plants to
contaminate wells of enemies
WORLD TRADE
CENTER
TWIN TOWER
ATTACK
Terrorist Attacks on the WTC Twin Towers
ANTHRAX -TAINTED LETTERS
INVISIBLE OR MICROSCOPIC
SIMPLE LAB TECHNIQUES REQUIRED
MAY NOT REQUIRE SOPHISTICATED APPARATUS
EASY TO MULTIPLY AND MAINTAIN
DIFFICULT TO TRACE
VERY DEADLY
DISADVANTAGES OF BIOLOGICAL WEAPONS
(FOR TERRORISTS)
HIGH RISK OF WORKER BEING CONTAMINATED
LIVING ORGANISM MAY BE DESTROYED WHEN
INCORPORATED INTO BOMBS AND MISSLES (BY HEAT)
PROBLEM OF DISPERSAL AND ATTACK, difficult to deploy
REQUIRES CONFIRMATION THAT STRAINS ARE
PATHOGENIC OR DISEASE CAUSING
PROBLEMS OF ACQUISITION FOR HIGHLY
RESTRICTED MICROBES
AND WHAT ABOUT ANTHRAX ?
Usually affects livestock
CAUSED BY A BACTERIUM , Bacillus anthracis
No proven
treatment
CAN KILL WITHIN WEEKS, FATAL IN ABOUT 30 %
OF CASES
Vaccine is available which can lessen severity of
disease
AND HOW ABOUT THE PLAGUE ?