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Applications of medical biotechnology
An introduction to
MEDICAL BIOTECHNOLOGY
WHAT IS MEDICAL OR RED
BIOTECHNOLOGY

?
Medical Biotechnology is the use of
living cells and cell materials to
research and produce pharmaceutical
and diagnostic products that help treat
and prevent human diseases.
BIOTECHNOLOGY CLASSIFICATION
Based on the field of
application biotechnology
can be classified in to
many…
RED BIOTECHNOLOGY
Or MEDICAL BIOTECHNOLOGY is
biotechnology applied to manufacture
pharmaceuticals like

enzymes, antibiotics and vaccines, and
its use for molecular diagnostic.

WHITE BIOTECHNOLOGY
Or INDUSTRIAL BIOTECHNOLOGY is
biotechnology applied to
industrial and other production
processes.

BIOTECHNOLOGY
GREEN
BIOTECHNOLOGY
Or AGRICULTURAL BIOTECHNOLOGY
is biotechnology applied to
agricultural processes and
products.

BLUE
BIOTECHNOLOGY

Or marine biotechnology is

marine and aquatic applications
of biotechnology
APPLICATIONS OF MEDICAL BIOTECHNOLOGY
1. PHARMACOLOGY
2. GENE THERAPY

3. STEM CELLS
4. TISSUE ENGINEERING
APPLICATIONS OF MEDICAL BIOTECHNOLOGY
1. PHARMACOLOGY
2. GENE THERAPY

3. STEM CELLS
4. TISSUE ENGINEERING
PHARMACOLOGY
1. INSULIN
PRODUCTION

Production of genetically engineered human insulin was one of the first
breakthroughs of biotechnology in the pharmaceutical industry.
Insulin was first produced in Escherichia coli through recombinant DNA
technology in 1978.
 PRINCIPLE:- Mass production of human proteins, vaccines, etc. by genetically
modifying bacteria or viruses.
PROCESS:- The human gene for insulin is placed into bacteria, are cultured and
allowed to produce insulin which is collected, purified and sold to diabetics
worldwide.




Grow bacteria that make the insulin protein
(fermentation)
Isolate the protein from all the other stuff that was in the
fermentation tank (purification)
Convert the insulin to its active form (processing)
2.HUMAN GROWTH HORMONE
Production of human growth hormone was first done in 1979 using
recombinant DNA technology.
scientists produced human growth hormone by inserting DNA coding for
human growth hormone into a plasmid that was implanted in Escherichia
coli bacteria.
This gene that was inserted into the plasmid was created by reverse
transcription of the mRNA found in pituitary glands to complementary DNA.
Prior to this development, human growth hormone was extracted from the
pituitary glands of cadavers, as animal growth hormones have no
therapeutic value in humans.
3.HUMAN BLOOD CLOTTING FACTOR
Production of human clotting factors was enhanced through
recombinant DNA technology.
Human clotting factor ix was the first to be produced through
recombinant DNA technology using transgenic Chinese hamster
ovary cells in 1986.

Plasmids containing the factor IX gene, along with plasmids with a
gene that codes for resistance to methotrexate, were inserted into
Chinese hamster ovary cells via transfection.
4.GENE PILL
1.
2.
3.
4.

Gene pill delivers DNA to Intestine
DNA is absorbed by gut cells
Protein drug is synthesized inside the cells
Protein drug is secreted into the blood
5. MONOCLONAL ANTIBODIES
(MAB)

They are so called because they are clones of an
individual parent cell.
 Remember, antibodies are specific proteins that
target pathogens invading our body.


1.
2.

3.

4.

Steps in making them:
Human antibody genes are put into a mouse.
Mouse is infected causing it to make human
antibody producing cells (B-cells).
These cells are removed from the mouse and
fused with a tumour cell.
Now we have a tumour cell that is constantly
producing antibodies and more cells like itself.
ADVANTAGES

This technology is used primarily to fight off cancer
cells as these monoclonal antibodies can be
“trained” to target markers that show up on cancer
cells.
 The mAbs will then destroy the cancer cell and go
looking for more.

SOME PROTEIN THERAPEUTICS MADE BY BIOTECHNOLOGY
AND ITS FUNCTIONS
APPLICATIONS OF MEDICAL BIOTECHNOLOGY
1. PHARMACOLOGY
2. GENE THERAPY
3. STEM CELLS
4. TISSUE ENGINEERING
GENE THERAPY
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease.
 It derives its name from the idea that DNA can be used to supplement or alter genes
within an individual's cells as a therapy to treat disease

 The most common form of gene therapy involves using DNA that encodes a functional,
therapeutic gene to replace a mutated gene.
Gene therapy is of two types , somatic gene therapy and germ line gene therapy.
GENE THERAPY FOR DISEASES
Gene Therapy has made important
medical advances in less than two
decades. Within this short time span, it has
moved from the conceptual stage to
technology development and laboratory
research to clinical translational trials for a
variety of deadly diseases. The most
notable advancements are the following:
GENE THERAPY FOR GENETIC DISORDERS

SEVERE COMBINED IMMUNE DEFICIENCY (ADA-SCID)
ADA-SCID is also known as the bubble boy disease.
 Affected children are born without an effective immune system and
will succumb to infections outside of the bubble without bone marrow
transplantation from matched donors.

 The therapeutic gene called ADA was introduced into the bone
marrow cells of such patients in the laboratory, followed by
transplantation of the genetically corrected cells back to the same
patients.
 The immune system was reconstituted in all six treated patients
without noticeable side effects, who now live normal lives with their
families without the need for further treatment.
CHRONIC GRANULOMATUS DISORDER (CGD)

CGD is a genetic disease in the immune system that leads
to the patients' inability to fight off bacterial and fungal
infections that can be fatal.
 Using similar technologies as in the ADA-SCID trial,
investigators in Germany treated two patients with this
disease, whose reconstituted immune systems have since
been able to provide them with full protection against microbial
infections for at least two years.
HEMOPHILIA
Patients born with Hemophilia are not able to induce blood
clots and suffer from external and internal bleeding that can
be life threatening.
The therapeutic gene was introduced into the liver of
patients, who then acquired the ability to have normal blood
clotting time.
GENE THERAPY FOR ACQUIRED DISEASES
Multiple gene therapy strategies have been developed to treat a wide
variety of acquired diseases like

Cancer
Parkinson's Disease
Huntington's Disease
Influenza
HIV
Hepatitis
APPLICATIONS OF MEDICAL BIOTECHNOLOGY
1. PHARMACOLOGY
2. GENE THERAPY

3. STEM CELLS
4. TISSUE ENGINEERING
STEM CELLS








A stem cell is a cell that has the potential to
become any cell type in the human body.
Everyone has stem cells, but they are very hard to
access.
The easiest place to get stem cells is from an
embryo.
Stem cells are introduced into a damaged area of
the body where, under the right conditions, will
replace the damaged area.
Principles

Application

Process

Stem cells are

The main areas where stem

Often times stem

introduced into a

cells have proven their

cells are grown in

damaged area of the

worth is in bone marrow

a lab first to

body where, under the

transplants, replacing

ensure the right

right conditions, will

damaged heart tissue after a

conditions and

replace the damaged

heart attack and replacing

then placed into a

area.

damaged nerve tissue which

sick person.

gives hope to anyone who

has had a spinal cord injury.


Stem cells are currently being tested to treat everything from
Crohn’s disease to baldness!



The main areas where stem cells have proven their worth is in

bone marrow transplants, replacing damaged heart tissue after a
heart attack and replacing damaged nerve tissue which gives hope
to anyone who has had a spinal cord injury.
STEM CELLS (SOURCES)
 Embryonic

 Infant


stem cells

and adult stem cells

Present in small numbers in



Peripheral blood



Skin epithelium



Umbilical cord blood





Bone marrow

Dental pulp of infant’s teeth

May be obtained by reprogramming somatic cells


Introduction of retroviruses carrying reprogramming genes into
fibroblasts
APPLICATIONS OF MEDICAL BIOTECHNOLOGY
1. BIOPROCESSING
2. PHARMACOLOGY
3. GENE THERAPY
4. STEM CELLS
5. TISSUE ENGINEERING
TISSUE ENGINEERING


A form of regenerative medicine, tissue
engineering is the creation of human tissue
outside the body for later replacement.



Usually occurs on a tissue scaffold, but can
be grown on/in other organisms
The technique to grow an ear follows the steps


1) taking a tiny piece of cartilage tissue,



2) dissolving away the white springy tissue to collect
the actual cells inside (the cells are microscopic and
trapped inside the white tissue called matrix)



3) expanding the number of cells by various
methods in the lab



4) placing that increased volume of cells on or in
mould that have a shape of an ear



5) implanting the new ear onto the patient.


Tissue engineers have created artificial skin,
cartilage and bone marrow.



Current projects being undertaken include creating
an artificial liver, pancreas and bladder.



Again, we are far from replacing a whole organ, but
just looking for “refurbishing” our slightly used ones
at the moment.

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Applications of medical biotechnology

  • 3. WHAT IS MEDICAL OR RED BIOTECHNOLOGY ?
  • 4. Medical Biotechnology is the use of living cells and cell materials to research and produce pharmaceutical and diagnostic products that help treat and prevent human diseases.
  • 5. BIOTECHNOLOGY CLASSIFICATION Based on the field of application biotechnology can be classified in to many…
  • 6. RED BIOTECHNOLOGY Or MEDICAL BIOTECHNOLOGY is biotechnology applied to manufacture pharmaceuticals like enzymes, antibiotics and vaccines, and its use for molecular diagnostic. WHITE BIOTECHNOLOGY Or INDUSTRIAL BIOTECHNOLOGY is biotechnology applied to industrial and other production processes. BIOTECHNOLOGY GREEN BIOTECHNOLOGY Or AGRICULTURAL BIOTECHNOLOGY is biotechnology applied to agricultural processes and products. BLUE BIOTECHNOLOGY Or marine biotechnology is marine and aquatic applications of biotechnology
  • 7. APPLICATIONS OF MEDICAL BIOTECHNOLOGY 1. PHARMACOLOGY 2. GENE THERAPY 3. STEM CELLS 4. TISSUE ENGINEERING
  • 8. APPLICATIONS OF MEDICAL BIOTECHNOLOGY 1. PHARMACOLOGY 2. GENE THERAPY 3. STEM CELLS 4. TISSUE ENGINEERING
  • 9. PHARMACOLOGY 1. INSULIN PRODUCTION Production of genetically engineered human insulin was one of the first breakthroughs of biotechnology in the pharmaceutical industry. Insulin was first produced in Escherichia coli through recombinant DNA technology in 1978.  PRINCIPLE:- Mass production of human proteins, vaccines, etc. by genetically modifying bacteria or viruses. PROCESS:- The human gene for insulin is placed into bacteria, are cultured and allowed to produce insulin which is collected, purified and sold to diabetics worldwide.
  • 10.    Grow bacteria that make the insulin protein (fermentation) Isolate the protein from all the other stuff that was in the fermentation tank (purification) Convert the insulin to its active form (processing)
  • 11. 2.HUMAN GROWTH HORMONE Production of human growth hormone was first done in 1979 using recombinant DNA technology. scientists produced human growth hormone by inserting DNA coding for human growth hormone into a plasmid that was implanted in Escherichia coli bacteria. This gene that was inserted into the plasmid was created by reverse transcription of the mRNA found in pituitary glands to complementary DNA. Prior to this development, human growth hormone was extracted from the pituitary glands of cadavers, as animal growth hormones have no therapeutic value in humans.
  • 12. 3.HUMAN BLOOD CLOTTING FACTOR Production of human clotting factors was enhanced through recombinant DNA technology. Human clotting factor ix was the first to be produced through recombinant DNA technology using transgenic Chinese hamster ovary cells in 1986. Plasmids containing the factor IX gene, along with plasmids with a gene that codes for resistance to methotrexate, were inserted into Chinese hamster ovary cells via transfection.
  • 13. 4.GENE PILL 1. 2. 3. 4. Gene pill delivers DNA to Intestine DNA is absorbed by gut cells Protein drug is synthesized inside the cells Protein drug is secreted into the blood
  • 14. 5. MONOCLONAL ANTIBODIES (MAB) They are so called because they are clones of an individual parent cell.  Remember, antibodies are specific proteins that target pathogens invading our body. 
  • 15.  1. 2. 3. 4. Steps in making them: Human antibody genes are put into a mouse. Mouse is infected causing it to make human antibody producing cells (B-cells). These cells are removed from the mouse and fused with a tumour cell. Now we have a tumour cell that is constantly producing antibodies and more cells like itself.
  • 16. ADVANTAGES This technology is used primarily to fight off cancer cells as these monoclonal antibodies can be “trained” to target markers that show up on cancer cells.  The mAbs will then destroy the cancer cell and go looking for more. 
  • 17. SOME PROTEIN THERAPEUTICS MADE BY BIOTECHNOLOGY AND ITS FUNCTIONS
  • 18. APPLICATIONS OF MEDICAL BIOTECHNOLOGY 1. PHARMACOLOGY 2. GENE THERAPY 3. STEM CELLS 4. TISSUE ENGINEERING
  • 19. GENE THERAPY Gene therapy is the use of DNA as a pharmaceutical agent to treat disease.  It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease  The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Gene therapy is of two types , somatic gene therapy and germ line gene therapy.
  • 20. GENE THERAPY FOR DISEASES Gene Therapy has made important medical advances in less than two decades. Within this short time span, it has moved from the conceptual stage to technology development and laboratory research to clinical translational trials for a variety of deadly diseases. The most notable advancements are the following:
  • 21. GENE THERAPY FOR GENETIC DISORDERS SEVERE COMBINED IMMUNE DEFICIENCY (ADA-SCID) ADA-SCID is also known as the bubble boy disease.  Affected children are born without an effective immune system and will succumb to infections outside of the bubble without bone marrow transplantation from matched donors.  The therapeutic gene called ADA was introduced into the bone marrow cells of such patients in the laboratory, followed by transplantation of the genetically corrected cells back to the same patients.  The immune system was reconstituted in all six treated patients without noticeable side effects, who now live normal lives with their families without the need for further treatment.
  • 22. CHRONIC GRANULOMATUS DISORDER (CGD) CGD is a genetic disease in the immune system that leads to the patients' inability to fight off bacterial and fungal infections that can be fatal.  Using similar technologies as in the ADA-SCID trial, investigators in Germany treated two patients with this disease, whose reconstituted immune systems have since been able to provide them with full protection against microbial infections for at least two years.
  • 23. HEMOPHILIA Patients born with Hemophilia are not able to induce blood clots and suffer from external and internal bleeding that can be life threatening. The therapeutic gene was introduced into the liver of patients, who then acquired the ability to have normal blood clotting time.
  • 24. GENE THERAPY FOR ACQUIRED DISEASES Multiple gene therapy strategies have been developed to treat a wide variety of acquired diseases like Cancer Parkinson's Disease Huntington's Disease Influenza HIV Hepatitis
  • 25. APPLICATIONS OF MEDICAL BIOTECHNOLOGY 1. PHARMACOLOGY 2. GENE THERAPY 3. STEM CELLS 4. TISSUE ENGINEERING
  • 26. STEM CELLS     A stem cell is a cell that has the potential to become any cell type in the human body. Everyone has stem cells, but they are very hard to access. The easiest place to get stem cells is from an embryo. Stem cells are introduced into a damaged area of the body where, under the right conditions, will replace the damaged area.
  • 27. Principles Application Process Stem cells are The main areas where stem Often times stem introduced into a cells have proven their cells are grown in damaged area of the worth is in bone marrow a lab first to body where, under the transplants, replacing ensure the right right conditions, will damaged heart tissue after a conditions and replace the damaged heart attack and replacing then placed into a area. damaged nerve tissue which sick person. gives hope to anyone who has had a spinal cord injury.
  • 28.  Stem cells are currently being tested to treat everything from Crohn’s disease to baldness!  The main areas where stem cells have proven their worth is in bone marrow transplants, replacing damaged heart tissue after a heart attack and replacing damaged nerve tissue which gives hope to anyone who has had a spinal cord injury.
  • 29. STEM CELLS (SOURCES)  Embryonic  Infant  stem cells and adult stem cells Present in small numbers in   Peripheral blood  Skin epithelium  Umbilical cord blood   Bone marrow Dental pulp of infant’s teeth May be obtained by reprogramming somatic cells  Introduction of retroviruses carrying reprogramming genes into fibroblasts
  • 30. APPLICATIONS OF MEDICAL BIOTECHNOLOGY 1. BIOPROCESSING 2. PHARMACOLOGY 3. GENE THERAPY 4. STEM CELLS 5. TISSUE ENGINEERING
  • 31. TISSUE ENGINEERING  A form of regenerative medicine, tissue engineering is the creation of human tissue outside the body for later replacement.  Usually occurs on a tissue scaffold, but can be grown on/in other organisms
  • 32. The technique to grow an ear follows the steps  1) taking a tiny piece of cartilage tissue,  2) dissolving away the white springy tissue to collect the actual cells inside (the cells are microscopic and trapped inside the white tissue called matrix)  3) expanding the number of cells by various methods in the lab  4) placing that increased volume of cells on or in mould that have a shape of an ear  5) implanting the new ear onto the patient.
  • 33.  Tissue engineers have created artificial skin, cartilage and bone marrow.  Current projects being undertaken include creating an artificial liver, pancreas and bladder.  Again, we are far from replacing a whole organ, but just looking for “refurbishing” our slightly used ones at the moment.