Lesson 12: The Gene Therapy (Stem Cells) 1.
Gene Augmentation Therapy suitable for the treatment of
used to treat diseases caused by a infectious diseases, cancer and
Gene Therapy mutation that stops a gene from inherited disease caused by
when DNA is introduced into a patient producing a functioning product, such as inappropriate gene activity
to treat a genetic disease a protein aim is to introduce a gene whose
the new DNA usually contains a adds DNA containing a functional product either:
functioning gene to correct the effects of version of the lost gene back into the o inhibits the expression of
a disease-causing mutation cell another gene
uses sections of DNA (usually genes) to the new gene produces a functioning o interferes with the activity
treat or prevent disease product at sufficient levels to replace the of the product of another
the DNA is carefully selected to correct protein that was originally missing gene
the effect of a mutated gene that is only successful if the effects of the basis of this therapy is to eliminate
causing disease disease are reversible or have not the activity of a gene that
the technique was first developed in resulted in lasting damage to the body encourages the growth of disease-
1972 (has had limited success in treating related cells
human diseases) Example:
may be a promising treatment option for can be used to treat loss of function Example:
some genetic diseases: muscular disorders such as cystic fibrosis by cancer is sometimes the result of the
dystrophy and cystic fibrosis introducing a functional copy of the over-activation of an oncogene
gene to correct the disease (gene which stimulates cell growth)
Two Different Types of Gene Therapy: by eliminating the activity of that
1. Somatic Gene Therapy oncogene through gene inhibition
transfer of a section of DNA to any therapy, it is possible to prevent
cell of the body that doesn’t produce further cell growth and stop the
sperm or eggs cancer in its tracks
effects of gene therapy will not be 2. Gene Inhibition Therapy
passed onto the patient’s children
2. Germline Gene Therapy
transfer of a section of DNA to cells
that produce eggs or sperm
Effects of gene therapy will be
passed onto the patient’s children
and subsequent generations
GENE THERAPY TECHNIQUES
3. Killing of Specific Cells HOW IS DNA TRANSFER DONE? a healthy and normal gene is inserted
suitable for diseases such as cancer a section of DNA or gene containing into a cell or tissue to replace a gene that
that can be treated by destroying instructions for making a useful protein is abnormal and thus, produces disease
certain groups of cells is packaged within a vector (usually a delivery of the gene can be difficult and
aim is to insert DNA into a diseased virus, bacterium or plasmid) viruses are commonly used to ‘carry’
cell that causes that cell to die the vector acts as a vehicle to carry the the normal gene into the cell
can be achieved in one of two ways: new DNA into the cells of a patient with
o the inserted DNA contains a a genetic disease USING STEM CELLS FOR GENE
“suicide” gene that produces once inside the cells of the patient, the THERAPY
a highly toxic product DNA or gene is expressed by the cell’s Stem Cells
which kills the diseased cell normal machinery leading to production have the ability to self-renew, which
o the inserted DNA causes of the therapeutic protein and treatment means that the need to provide repeated
expression of a protein that of the patient’s disease administrations of gene therapy can be
marks the cells so that the reduced or possibly even eliminated
diseased cells are attacked
by the body’s natural Hematopoietic Stem Cells
immune system an ideal choice because they can easily
be removed from the blood, bone
essential with this method that the marrow or umbilical cord
inserted DNA is targeted appropriately not as difficult to identify and isolate as
to avoid the death of cells that are other stem cells and can be more readily
functioning normally CHALLENGES OF GENE THERAPY coaxed to differentiate in a laboratory
Delivering the gene to the right place setting before injection into the patient
and switching it on
Avoiding the immune response NOTE: Other stem cells that suggest promise
Making sure the new gene doesn’t for gene therapy include myoblasts and neural
disrupt the function of other genes stem cells.
The cost of gene therapy
Myoblasts
UNDERSTANDING GENE THERAPY work particularly well for injection into
Gene Therapy muscle tissue because they readily join
involves inserting genes into a person’s with other muscle fibers and therefore
cells with the aim of providing treatment integrate well into the network of
for a particular disease muscle fibers
the use of a person’s own cells to
provide a therapeutic effect Neural Stem Cells
appear to hold potential for treating give hope of healthy life to cancer
gliomas: a difficult type of brain tumor patient
to treat for certain disease that do not have any
cure except gene therapy, it could save
LIST OF THE PROS OF GENE THERAPY many lives
provides hope for those who may not DISADVANTAGES OF GENE
have had any in the past THERAPY
could change the perspectives that the genetic testing, screening and
people have about disease research in finding the availability of
could offer the potential of new certain gene is very controversy
discoveries may increase rate of abortion if prenatal
could be used in different ways to test regarding baby with genetic disease
improve life is done
does not just apply to human treatment the cost is very high and the patient
options might need an insurance to cover the
allows us to use technology to improve treatment
the quality of life for people cosmetic industry may monopo-lized
allows us to treat the “untreatable” this gene therapy if it is used in
diseases enhancing beauty and in vanishing the
aging effect, rather than used for
LIST OF THE CONS OF GENE THERAPY treatment of a disease
does not have a reliable delivery method
an expensive procedure
requires ongoing treatments to be
effective
may not be able to adapt to a changing
world
might only delay the inevitable
will shift society toward new
polarization
could change the way we think about
competition
ADVANTAGES OF GENE THERAPY
give a chance of a normal life to baby
born with genetic disease
