The Aspects of

Gene Therapy
Reporters:
Sarbues, Aina Angela
Sonza, Francy Gwyn Beatriz
Toledo, Andre
Gene Therapy

It is a medical field which focuses on the

utilization of the therapeutic delivery of
nucleic acid into a patient’s cells as a drug to
treat disease. Gene therapy is a potential
method to either treat or cure genetic-related
illness.

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Human Gene
Therapy
Human Gene Therapy was
first realized in 1971
It was during this year when the first recombinant DNA
experiments were planned. Foreign DNA is inserted into a
patient’s tissue with hopes to eradicate a targeted disease. It was
actually inspired by the success of recombinant DNA
technology which occurred over the last 20 years. Without a
doubt, gene therapy is the most promising yet possibly
unfavorable medical field being studied.

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Basic Process (Fliesler, 2017)
⦁ Replacement of mutated
gene that causes disease
with a healthy copy of
gene
⦁ Inactivation of a mutated
gene that is functioning
improperly
⦁ Introducing a new gene
into the body to help fight
a disease
In general, a gene cannot be
directly inserted into a human
gene or cell. A gene is inserted
into another gene using a carrier
or a vector. At present, the most
common type of vectors are
viruses that have been genetically
changed to carry normal human
DNA. Viruses have evolved a way
of encapsulating and transporting
their genes to human cells in a
pathogenic manner.
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Two Types of 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.
Germ-line Gene Therapy which
involves the genetic modification
of germ cells or the origin cells
that will pass the change on to the
next generation.
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2
Stem Cell Gene
Therapy
Stem cells are mother cells that have the Characteristic: has the
potential to become any type of cell in the ability to self-renew or
multiply while maintaining
body. the potential to develop into
Two sources of stem cells:
other types of cells

⦁ Embryonic stem cells are derived from a four- or five-day-old

human embryo that is in the blastocyst phase of development. The
embryos are usually extras that have been created in IVF clinics
where several eggs are fertilized in a test tube then implanted into
a woman.
⦁ Somatic stem cells are cells that exist throughout the body after
embryonic development and are found inside of different types of
tissue. These stem cells have been found in tissues such as the
brain, bone marrow, blood, blood vessels, skeletal muscles, skin,
and the liver. They remain in a non-dividing state for years until
activated by disease or tissue injury. 8
“
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