Scientific Proposal

1. PARKINSON DISEASE- SPECIFIC
INDUCED PLURIPOTENT STEM
CELLS
Sara C. Díaz-Méndez
RISE Program
Enrique Rodriguez-Borrero,

2. PARKINSON'S DISEASE (PD)

3. Parkinson's is a
progressive movement
disorder that usually
strikes after age 50.
Parkinson’s involves
the malfunction and
death of vital nerve
cells in the brain, called
neurons.
The neurons that die
in Parkinson's Disease
connect a structure in
the brain called the
substantia nigra to
another structure called
the striatum.

4. Alternative Approaches
for treating Parkinson's
disease:
• For many years, doctors have
treated Parkinson's disease
patients with the drug
levodopa, which the brain
converts into dopamine.
Although the drug works well
initially, levodopa eventually
loses its effectiveness, and
side-effects increase.
 Surgery, such as deep brain
stimulation with implanted
electrodes, is used to treat
advanced symptoms. But it
does not slow down or
reverse the damage to nerve
cells in the brain. Over time,
the symptoms often get worse
despite treatment.
 Pluripotent embryonic stem
cells have been recently used
by scientists and are under
investigation.

5. WHAT ARE PLURIPOTENT STEM
CELLS?
 Stem cells are defined by two
properties. First, they can ‘self-renew,’ that
is, they can divide and give rise to more
stem cells of the same kind. Second, they
can mature or ‘differentiate’ into
specialized cells that carry out a specific
function.

6. PROBLEM
• Recent studies in rodents with
embryonic stem cells to replace
dopamine producing neurons in the
brain have develop tumors as a side
effect.

7. CHALLENGE
 Use a different type of pluripotent stem cells that
won’t be rejected from a person's body due to their
immune system.
 Many scientists and members of the public have
ethical issues with the use of pluripotent stem cells
from human embryos or fetal tissues.

8. HYPOTHESIS
Using pluripotent cells from the patient’s skin to
produce pluripotent embryonic like cells and from these
cells generate the dopamine producing neurons that
will reduce the risk of immunological rejection of the
new neuron cells.

9. METHODOLOGY
1. I will use a large number of rodents that will allow my
results to be statistically significant. First I will locate the
midbrain area in rodents involved in dopamine
production.
1. Induced pluripotent stem (iPS) cells are going to be made
from a rodent’s skin cells in the lab, and then used to
make dopamine-producing neurons.
- To make a specific type of cell in high numbers,
scientists expose the stem cells to a cocktail of chemicals
that mimic what they would experience during normal
development. The resulting cells also carry more of the
molecular markers that characterize dopamine-producing
cells in the brain.

10. METHODOLOGY
• 3. Surgically I will introduced the generated
dopamine producing neurons in the midbrain area,
which is where dopamine neurons are normally
located. Since a single, well-defined type of cell is
affected, it is possible to treat Parkinson’s by
replacing the lost nerve cells with healthy new ones.
• 4. Their physical and motor skills will be observe to
evaluate the progress of the disease. Laboratory
blood analysis will be performed to monitor the
progress of the disease.

11. FUTURE INVESTIGATION
• Studies will be needed to make sure the cells are
safe and would not cause tumors in the brain.

12. EXPECTED OUTCOME
• This will improve the quality of life of elderly people
affected by Parkinson's Disease and extend their life
expectancy. It will also reduce the cost of caring of
elderly people.

13. REFERENCES:
• "All About Parkinso's Disease." . Marc, 25 Apr. 2014. Web. 21 June 2014.
<http://coloradodementia.org/category/parkinsons-disease-2/>.
• "Parkinson's Disease." . NIH Senior Health, 1 June 2012. Web. 1 June
2014.
<http://nihseniorhealth.gov/parkinsonsdisease/whatcausesparkinsonsdise
ase/01.html>.
• "STEM CELL RESEARCH AND PARKINSON’S DISEASE." . Parkinson
Society Canada, 1 May 2009. Web. .
<http://www.parkinson.ca/atf/cf/%7B9EBD08A9-7886-4B2D-A1C4-
A131E7096BF8%7D/stem%20cells%20-%20en.pdf>.
• "Reprogramming: how to turn any cell of the body into pluripotent stem
cell ." . Euro Stem Cell , 14 Dec. 2012. Web. .
<http://www.eurostemcell.org/factsheet/reprogramming-how-turn-any-cell-
body-pluripotent-stem-cell>.
• "Stem Cells and Diseases." . National Institude of Health, 1 Dec. 2013.
Web. . <http://stemcells.nih.gov/info/pages/health.aspx>.