GENE THERAPY

UHA PRODUCTIONS

WHAT IS GENE THERAPY?

Definition: A transplantation of normal genes in place of missing or defected genes in order to

correct gene disorders.
If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to
introduce a normal copy of the gene to restore the function of the protein.

HOW DOES IT WORK?

Researchers typically add new genes by using a virus to carry the genetic cargo into the cell,
considering that viruses have evolved into doing with their own genetic material.
A gene that is inserted directly into a cell usually does not function. The gene or virus to fight the
gene that is not working are modified so they cant cause disease when used in people.

Gen
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Detailed
Explanation:
experimental technique that uses genes to prevent disease this technique
might allow doctors to treat disorders by inserting certain genes to a patients
cells apposed to resulting to drugs or surgery a way to fix genetic problems
at the roots adding a correct copy of a defective genes to a patients genes to
fix their problem and allow their damaged tissue/organs to work properly

WHEN DID THIS START?

On September, 14, 1990, a 4 year old girl was diagnosed with ADA deficiency which left her
defenceless against protection from infections. Her white blood cells were taken from her, and
the normal genes for making adenosine deaminase were added, and then the corrected cells
were then re injected into her.

Introduced by:
Dr. W. French Anderson

The Problems
so far has seen limited success
hard to produce genes into cells of the body and keep them working
financial concerns

GENE DELIVERY AND ACTIVATION:

for some problems, it will work if a normal cell can be delivered to a large number
of cells in a tissue (millions)
the cells have to be correct and put into the correct tissue
once gene reaches destination it must turn on and stay on which is problematic
because cells have the habit of shutting down overly active genes
delivering genes to the wrong tissue could increase problems for a person

WHAT HAPPENS IF GENE THERAPY GOES

WRONG?
Unwanted immune system reaction:
Your body's immune system may see the newly introduced viruses as intruders and
attack them. This may cause inflammation and, in severe cases, organ failure.

Targeting the wrong cells:

Because viruses can affect more than one type of cells, it's possible that the altered
viruses may infect additional cells not just the targeted cells containing mutated
genes. If this happens, healthy cells may be damaged, causing other illness or diseases,
including cancer.

Infection caused by the virus:

It's possible that once introduced into the body, the viruses may recover their original
ability to cause disease.

Possibility of causing a tumor:

If the new genes get inserted in the wrong spot in your DNA, there is a chance that the
insertion might lead to tumor formation. This has occurred occasionally in some clinical
trials

WHY THIS IS UNCOMMON

Commercial viability:
genetic disorders that could be treated by gene therapy are rare
could be a life saving solution for the patients but high developing costs prevents
the lack of purchase by pharmaceutical companies
developing a new therapy is expensive
developers may never earn money from treating rare disorders
some patients may never be able to afford them either
some diseases that can be treated with gene therapy ex. cancer, are much more
common
more promising gene therapy approaches are individualized to each patient

GENE THERAPY

BIBLIOGRAPHY
What is gene therapy U.S National Library of Medicine. Oct 2013. Web. Nov 17. 2015.
Gene therapy Genetic science learning center. 2015. Web. Nov 17.2015.
"How Does Gene Therapy Work?" Scientific American Global RSS. 13 May 2008. Web. 6 Nov.
2015.

"Gene Therapy - A Revolution in Progress: Human Genetics and Medical Research." Gene
Therapy - A Revolution in Progress: Human Genetics and Medical Research. Web. 6 Nov.
2015.

"Google." Google. Web. 6 Nov. 2015