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Stem Cell

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Stem Cell

Objectives
To know:
 What is Stem cell
 What is Gene Therapy
 What are the two types of Gene Therapy
 What are the techniques of Gene therapy
 What are the advantages and disadvantages of Gene
Therapy
What is Stem Cell?
Stem cells are biological cells that can 
differentiate into other types of cells and can 
divide to produce more of the same type of
stem cells. They are found in 
multicellular organisms.

https://en.wikipedia.org/wiki/Stem_cell
What is gene therapy?
Gene therapy is an experimental technique
that uses genes to treat or prevent disease. In
the future, this technique may allow doctors
to treat a disorder by inserting a gene into a
patient’s cells instead of using drugs or
surgery.

https://ghr.nlm.nih.gov/primer/therapy/genetherapy
There are two different types of gene therapy
depending on which types of cells are treated:

 Somatic gene therapy: transfer of a section of DNA to


any cell of the body that doesn’t produce sperm or eggs.
Effects of gene therapy will not be passed onto the
patient’s children.
 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.

https://www.yourgenome.org/facts/what-is-gene-therapy
Somatic gene therapy

http://www.open.edu
/openlearn/science-
maths-
technology/science/b
iology/gene-
therapy/content-
section-3
Germline gene therapy

http://science.sciencem
ag.org/content/348/624
1/1325.1
Gene therapy techniques

Gene augmentation therapy


Gene inhibition therapy
Gene augmentation therapy
 This is used to treat diseases caused by a mutation that
stops a gene from producing a functioning product, such as
a protein?.
 This therapy adds DNA containing a functional version of
the lost gene back into the cell.
 The new gene produces a functioning product at sufficient
levels to replace the protein that was originally missing.
 This is only successful if the effects of the disease are
reversible or have not resulted in lasting damage to the
body.

https://www.yourgenome.org/facts/what-is-gene-therapy
Gene augmentation therapy

https://www.yourgenome.org/facts/what-is-gene-therapy
Gene inhibition therapy
 Suitable for the treatment of infectious diseases, cancer
and inherited disease caused by inappropriate gene
activity.
 The aim is to introduce a gene whose product either:
 inhibits the expression of another gene
 interferes with the activity of the product of another
gene.
 The basis of this therapy is to eliminate the activity of a
gene that encourages the growth of disease-related cells.

https://www.yourgenome.org/facts/what-is-gene-therapy
Gene inhibition therapy

https://www.yourgenome.org/facts/what-is-gene-therapy
Advantages of Gene Therapy
 One of the benefits of somatic gene therapy is that it can be utilized
in a way that can possibly cure patients that are severely ill. It can
also prevent the onset of devastating illnesses.
 Although this is experimental,  medical professionals generally use
the best treatment methods available.  Using this process, it
promotes the freedom of scientific inquiry and the freedom of
knowledge.
 Gene therapy, when successful, can have a number of advantages in
comparison to drug therapy .For example, this  therapy can provide
a cure rather than ease the patient's symptoms.

https://genetherapyinthefuture.weebly.com/advantages-and-disadvantages.html
Disadvantages of Gene Therapy
 Due to the fact that this is still a new scientific breakthrough, the safety concerns
outweigh quite a bit of the procedures. The current  understanding of gene therapy
is based on theory rather than fact.
 There are numerous ethical concerns, as well as religious and moral concerns.
 Concerning the vector: once is is administered, there is no way for certain to tell if
the vector will travel to the intended location. This creates even more damage to
the genetic structure and cause more damage to the patient.
 In clinical trials already carried out the effects of the treatment have only been
short-lived. To achieve long term results much more research is needed.

https://genetherapyinthefuture.weebly.com/advantages-and-disadvantages.html

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