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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 a 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.
• Stem cells can become cells of the blood, heart, bones, skin,
muscles, brain, among others
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Timeline of the development of gene therapy:

• 1960s: Research began on stem cells taken from adult

tissue.
• 1968: Adult stem cells were used to treat an
immunodeficient patient.
• 1998: US scientists grew stem cells from human embryos
and germ cells, establishing cell lines still is use today

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Timeline of the development of gene therapy:

• 2001: Embryonic stem cell turned into a blood cell.

• 2004: South Korean scientists cloned 30 human embryos
and developed them over several days.
• 2005: The same team of South Korean scientists
developed stem cells tailored to match individual patients.

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Timeline of the development of gene therapy:

• 2006: Japanese scientists identified conditions that would

allow some specialized adult cells to be “reprogrammed”
genetically to assume a stem cell-liked state.
• 2007: US scientists extracted stem cells from cloned
monkey embryos and developed them into mature heart
and nerve cells in the laboratory.

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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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• 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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 Human diseases that can be treated by stem
cell therapy:
• Diabetes
• Rheumatoid arthritis
• Neurodegenerative diseases (Parkinson’s, Alzheimer's, &
amyotrophic lateral sclerosis)
• Osteoarthritis
• Stroke and traumatic brain injury
• Spinal cord injury
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 Bone Marrow Transplant
• Method of stem cell therapy that has been used for many years
with controversy.
• This process involves harvesting stem cells from the patient’s
bone marrow, typically from the pelvis, using large needle that
reaches the core of the bone.

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 Somatic cell nucleus transfer
• Another method that consists of taking a enucleated female ovum
(egg cell) and implanting a donor nucleus from a somatic (body)
cell.
• The ovum is then stimulated to divide as if it has been fertilized,
thus creating a human embryo.
• This embryo would then consists of pluripotent stem cells that
generally match the donor’s DNA from which they came.

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