Stem cell technologies Current state Future promise

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Stem cell technologiesCurrent stateFuture
promise

• Where UCI fits in to the Post Prop. 71 world of
  biotechnology development in California

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Proposition 71 Purpose and Intent

• Create California Institute for Regenerative
  Medicine.
• Oversight by Independent Citizens Oversight
  Committee (ICOC).
• Support the development of technologies involving
  pluripotent stem cell and progenitor cell.
• Prohibit human reproductive cloning

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Proposition 71 Funds

• Funding details
• Authorizes an average of 295 million per year in
  bonds over a 10-year period, 3 billion total
• In any year, no more than 350 million in bonds
  to be issued
• If less than 350 million is issued in any year,
  the remaining permitted amount may be carried
  over to one or more subsequent years

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Proposition 71 Research facilities

• Research facilities that can be used for research
  on non-approved lines are a priority.
• Accordingly, up to 10 of the funds will be
  used to build scientific and medical research
  facilities of nonprofit entities.
• These are to be constructed in the first five
  years, with priority given to facilities that can
  be brought on line within the first 2 years.
• Funding through Prop. 71 for development of
  facilities requires a 20 match.

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Proposition 71 Oversight

• The ICOC must meet in an open meeting format.
• Closed sessions may be conducted when discussing
• Matters involving information relating to
  patients or medical subjects. Matters involving
  confidential intellectual property or work
  product
• Matters involving prepublication, confidential
  scientific research or data
• Matters concerning the appointment, employment,
  performance, compensation, or dismissal of
  institute officers and employees

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Proposition 71 Scientific and Medical Working
Groups

• Scientific and Medical Research Funding Working
  Group.
• Scientific and Medical Accountability Standards
  Working Group.
• Scientific and Medical Research Facilities
  Working Group.

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A primer on Human Embryonic Stem Cells
Blastocyst - from In Vitro Fertilization Clinic
Blueprint cells
Inner Cell Mass (Stem Cells)
A Blastocyst is a hollow ball of cells with a
small clump of stem cells inside
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Human Embryonic Stem Cells
Pipette
Blueprint cells
Stem Cells
To remove the stem cells, the Blastocyst is
opened and the stem cells removed with a pipette
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Human Embryonic Stem Cells
Pipette
Blueprint cells
Stem Cells
Stem Cells
To remove the stem cells, the Blastocyst is
broken open and the stem cells removed with a
pipette(an ultra thin glass tube)
The stem cells are placed in a dish and are fed
and cared for (each blastocyst 1 stem cell
line)
Pipette
Petri Dish
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Growth factors
Chemical cues
Petri Dish
Stem Cells
Pancreatic Islet
Muscle cell
Neuron
Different chemicals / molecules are added to the
stem cells to make them become specific types of
cells.
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Somatic Cell Nuclear Transfer or Therapeutic
Cloning
Donor Egg
Skin Cell
Nucleus (DNA)
Nucleus (DNA)
Needle
Needle
Stem Cells
Chemicals and Growth Factors
Pancreatic Islet
Neuron
Muscle cell
Dividing cells
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The vision for UCIs Stem Cell Center Stem
cell therapies for neurological disorders

• Brain and spinal cord injury.
• Stroke.
• Neurodegenerative diseases
• Parkinsons Disease
• Huntingtons Disease
• Alzheimers Disease
• Multiple Sclerosis
• Lou Gerhigs Disease (ALS)

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Neurological disorders involve the loss of
particular cell types in the nervous system

• Brain and spinal cord injury and stroke (loss of
  nerve cells and myelin-forming oligodendrocytes).
• Neurodegenerative diseases
• Parkinsons Disease (loss of dopamine-containing
  nerve cells in the brainstem).
• Huntingtons Disease (loss of nerve cells in the
  striatum).
• Alzheimers Disease (loss of nerve cells in the
  cerebral cortex).
• Multiple Sclerosis (loss of myelin-forming
  oligodendrocytes).
• Lou Gerhigs Disease-ALS (loss of motor neurons
  from the spinal cord).
• The vision To use embryonic stem cells to
  restore the cells that are lost as a result of
  injury or neurodegenerative diseases.

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Goal 1 to make stem cells into nerve cells
Stem Cells
Blastocyst -
Pipette
Blueprint cells
Petri Dish
Stem Cells
Blueprint cells
Stem Cells
The stem cells are treated with factors to cause
them to differentiate into particular cell types
Stem cells differentiated into neurons
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Goal 2 To discover how to make stem cells
integrate into neural circuits.
Oligodendrocyte
Nerve cell (neuron)
More research is needed to find the ways to
actually use stem cells for therapeutic
applications.
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At the RIRC, a therapy is being developed to use
stem cells to replace myelin-forming cells
Myelin-forming cells (oligodendrocytes) die as a
result of spinal cord injury, resulting in the
loss of myelin (insulation) from nerve
fibers. An important potential therapeutic
strategy Replace myelin-forming cells using
stem cells that differentiate into
oligodendrocytes.
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RIRC scientists have succeeded in developing ways
to produce oligodendrocytes from human ES cells
and have shown that they can restore myelin after
spinal cord injury in experimental animals.
Stem cells are first differentiated to the neural
lineage.
Then to Oligodendrocyte Precursors
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But do these cells have the potential to form
tumors over longer periods of time?
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Embryonic Stem Cells

• They can generate large quantities of tissue
  rapidly

Human Embryonic Stem Cells
2. They can become any cell in the body
Embryonic stem cells
Brain
Heart
Fat
Bone marrow
Cartilage
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Developing facilities for stem cell research

• It is likely that research involving non-approved
  stem cell lines will be prohibited in facilities
  that were built with NIH support, or that contain
  NIH-supported core equipment.
• In recognition of this, up to 10 of Prop. 71
  funds will be used to build scientific and
  medical research facilities.
• Funding through Prop. 71 for development of
  facilities requires a 20 match.

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Proposition 71 Funds

• Indirect costs limited to 25 of a research
  award
• Excluding amounts included in a facilities
  award
• Indirect cost limitation may be increased by
  amount the grantee provides in matching funds
• Must be in excess of 20 of the grant amount