GENE THERAPY (STEMCELLS)

OBJECTIVES
To have a better view and knowledge
about the Modern Biotechnology
Explain the development of gene surgery
and gene theraphy to cure genetic diseases.
Application of Modern Biotechnology
 WHAT IS GENE?
A gene is the basic physical and functional unit
of heredity, Genes are
made up of DNA some
genes act as instructions
to make molecules
called protein.
 Contents
What is Gene Theraphy
and Stem Cell

Relationship of Cell and

Gene
Working with Stem
Cells

Purpose
 Is an experimental technique
that uses genes to treat or
prevent disease. This technique
may allow the doctors to treat
disorder by inserting gene into
patient’s cells instead of using
drug or surgery.
 HISTORY OF
GENE THERAPHY
 The concept of Gene Therapy arose
during the 1960s and 1970s and is still in
its infancy, meaning there is a paucity of
reliable, long-term data on the safety and
efficacy of this therapy.
 In the 1980’s scientist inserted human
genes into bacteria cell.
Then the bacteria cell transcribed and
translated the informaton into a protein.
Then they introduced the protein into
human cells.
STEM CELLS
 What is Stem Cells?
Stem Cell is a cell with the unique
ability to develop into specialized cell
types in the body. In some cases they also
have the ability to repair damaged tissues.
HISTORY OF STEM
CELLS
 Origin of the term Stem Cell

● Haeckel 1868
● Describe the origin of
Multicellular organism
from Unicellular
organism
 Alexander Maksimov
 Russian histologist who first
coined the term “stem cell” to
describe something he observed
about blood, in 1908.
 Who developed and introduced a
unitarian theory of hematopoiesis,
a theory upon which our present
concept of blood cells’origin and
differentiation is based.
 RELATIONSHIP OF
CELL TO GENE THERAPHY
 WHAT IS CELLS?
The smallest structural and
functional unit of
all living organism.

Building block of life.

 FUNCTONS OF CELL
- Provide structure for the body
- Take in nutrients from the food
- Converts those nutrients into energy
- Carry out specialized functions
Is the process of genetically
modifying sperm or
egg cells to create a new
offspring.
Affects only the targeted cells
in the patient, and is not
passed to future generations
Recombinant Viruses
Naked DNA / DNA Complexes
Working with Stem
Cells
 Stem Cells are unspecialized cells that develop into the
speciliazed cells that make different types of tissues in
the human body, characterized by the;

● Ability to renew themselves through mitotic cells

division. (self- renewal)

● Differentiating into a diverse range of specialized cell

types. (differentiation)
Classification of
Stem Cells
 Totipotent Stem Cells
It has the total potential to give rise to all
types of cells
Totipotent is the capacity of a single cell
to divide and differentiate into cell types.
 Pluripotent Stem Cells

Is the ability of the cells to produce

any type o cells in the organism,
they are derived from the mouse
embryo.
Multipotent Stem Cells

Means to those cells that can only

give rise to cells of the tissue from
which are they are isolated.
 Unipotent Stem Cell
The ability to only produce cells of
their own, but have the property of
self-renewal required to be labeled a
stem cell.
 Oligopotent Stem Cells
Means those cells that can
differentiate into only a few cell
types,like lymphoid or myeloid
stem cells.
Sources of Stem
Cells
 Embryonic Stem Cells

Derived from the embryos

Self replicating pluripotent cells that

are potentially immortal.
 Adult Stem Cell
 Undifferentiated totipotent or multipotent cells

 Primary roles are to maintain and

repair the tissues in which they are found
 Advantage of Gene Therapy
● Provides an opportunity for affected individuals to
lead a life that is “normal”.

● Removing healthy blood-forming stem cells from

the body

● inserting a gene that makes these cells more

resistant
● Preventing cancer cells from developing
new blood vessels, or angiogenesis

● It would create a new field of medicine.

● Gene therapies aren’t limited to humans.

 Disadvantage of Gene Therapy
● Response the body's immune system may see the newly introduced
viruses as intruders and attack them resulting in inflammation, toxicity,
as well as organ failure in severe cases.

● DNA mutations The new gene might be inserted in the wrong location
in the DNA

● Viral spread Because viruses can affect more than one type of cell, it
is possible that the viral vectors may infect cells beyond just those
containing mutated or missing genes.
● Reversion of the virus to its original form once
introduced into the body, the viruses may recover
their original ability to cause disease.

● Gene therapy is that, like any other medical

procedure, it may not work
 Advantage of Stem Cells
● It provides great potential for discovering treatments and cures

● Limbs and organs could be grown in a lab from stem cells and then used in
transplants or to help treat illnesses.

● Scientists and doctors will be able to test millions of potential drugs and
medicine, without the use of animals or human testers.
 Disadvantage of Stem Cell
● The use of embryonic stem cells for research involves the destruction
of blastocysts formed from laboratory-fertilized human eggs.

● Like any other new technology, it is also completely unknown what

the long-term effects of such an interference with nature could
materialize.

● most adult stem cells is that they are pre-specialized, for instance,
blood stem cells make only blood, and brain stem cells make only
brain cells
A shanti De Silva
The first gene therapy
case was performed on
September 14, 1990

She was treated for SCID (Severe Combined Immunodefiency)

M alachi Perfect
For Two
Reasons

He was diagnosed with Spinal Muscular Atrophy (SMA) type 1,

the most severe form of disease.
 B renden Whittaker

Gene therapy
was a
‘last shot’

Brenden has struggled with chronic granulomatous disease, or CGD, a rare

immune disorder that affects the function of infection-fighting white blood
cells called neutrophils.
B
runel Etienne New Gene Therapy
shows Promise For
Patients with Sickle
Cell Disease
Bartido, Lea Nicole Geres, Celeste
Carillo, Jeffrey Taladua, Trisha Marie