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Gene Therapy

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Gene Therapy 1
2/8/20XX
Medical science has detected many human
diseases related to defective genes. These
types of diseases are not curable by
traditional methods like taking readily
available medicines. Gene therapy is a
potential method to either treat or cure
genetic-related human illness.
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• Human gene therapy was actually first realize in
1971 when the first recombinant DNA experiments
were planned. It can be simply viewed as insertion
foreign DNA into a patients' tissue that hope to
successfully eradicate the targeted disease.
• Without a doubt, gene therapy is the most promising
yet possibly unfavorable medical field being studied.

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Basic Process
• Replacement of mutated gene that causes disease
with a healthy copy of the gene.
• Inactivation of mutated gene that is functioning
improperly
• Introducing s new gene into the body to help fight a
disease
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Two types of Gene Therapy
• The idea of gene therapy is based on correcting
a disease at its root; fixing the abnormal genes
that appear to certain diseases.
• Somatic Gene Therapy
• Germ-line Gene Therapy

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Somatic Gene Therapy
• Somatic Gene Therapy involves the manipulation of
genes in cells that will be helpful to the patient but
not inherited to the next generation
• Affects only the targeted cells in the patient.
• Short-lived because the cells of most tissues
ultimately die and are replaced by new cells.
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Germ-line Gene Therapy
• Germ-line Gene Therapy which involves the
genetic modification of germ cells or the origin
cells that will pass the change on the next
generation.
• Controversy
• Some people view this type of therapy as unnatural, and liken it to
“playing God”

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Stem Cell Gene Therapy
• Stem cells are mother cells that have the potential
to become any type of cell in the body.
• One of the main characteristics of stem cells is their
ability to self-renew or multiply maintaining the
potential to develop into other types of cells.

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Stem Cell Gene Therapy
•Stem cells can become cells of the
blood, heart, bones, skin, muscles,
brain, among other types of cell.

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Stem Cell Gene Therapy
• Stem Cells Therapy, also known as regenative
medicine, promotes the repair response of diseased,
dysfunctional or injured tissue using stem cells or
their derivates.
• The cells are manipulated to specialize into specific
type of cells such as heart muscle cells or blood
cells.
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•Stem cells are derived from
different sources. Two of which
are
•Embryonic Stem Cells
•Somatic Stem Cells
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Stem cells are derived from different sources. Two of which are;

• Embryonic Stem Cells – ESC are derived from a four-or five-day-


old human embryo that is in the blastocyst phase development. The
embryos are usually extras that have been created in IVF (in vitro
fertilization) clinics where several eggs are fertilized in a test tube then
implanted into a woman

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• Embryonic stem cells (ESCs) are found in the inner cell mass of
the human blastocyst, an early stage of the developing embryo
lasting from the 4th to 7th day after fertilization. In normal
embryonic development, they disappear after the 7 th day, and
begin to form the three embryonic tissue layer.

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• Somatic stem cells – SSC are cells that exist
throughout the body after embryonic development
and are found inside of different types of tissue.
• They are remain in a non-dividing state for years until
activated by disease or tissue injury.

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